Archive for the ‘Crispr’ Category

by Tyler English | published Dec. 11th, 2019

illustration by Darius Serebrova

Ever wonder where those neon yellow, green, blue and pink fish came from? You know, the ones that have all the matching accessories: tanks, decorations, rocks and their own special ultraviolet light? Well, as it turns out, a team ofscientists in Singapore were the first ones to genetically modify fish to glow in such a way.

Genetic editing in small animals and plants has been aroundsince the 1970s, according to Synthego, a company that providesgeneticallyedited stem cells. Starting with plants and bacteria, scientists began to explore the realm of DNA and genetics. As their understanding of the proteins grew, so did their curiosity.

When scientists learned how to modify the genes of small, simple organisms, they began to wonder, "How could this be applied to humans?"

The scientific community is stirring with the emergence of CRISPR DNA,more specifically known as the CRISPR-Cas9 protein.CRISPR stands forClustered Regularly Interspaced Short Palindromic Repeats.CRISPR is a faster, cheaper and more accurate way of editing the genome, according to theNational Institute of Health.By sending in two different pieces of CRISPR DNA,scientists are able to modify genes. To do so, theycutout areas of genes that aren'tperforming how they should be or as they're expected to.

Dr. Sandi Connelly, a principallecturer in the Thomas H. Gosnell School of Life Sciences, explained how DNA works and what the CRISPRCas-9 protein actually does. Connelly compared DNA to a street of houses each person has different foundations that sprout out different and unique homes.

CRISPR is a piece of DNA, and we [scientists] attach to it an enzyme ...it cuts the DNA at a very specific place like a pair of scissors, Connelly said. When we look at CRISPR, typically we look at CRISPR Cas-9."

Whereas CRISPR is the DNA itself, Cas-9 is the enzyme, a specialized protein that splits the DNA.Connelly said that this allows for both the CRISPR DNA and the original DNA to stick together like magnets. However, due to the specificity of this technique, scientists need to know where in the DNA they'relooking.

Using those same enzymes, we can cut [and] place back inthe good gene, Connelly said.

Now, this technique would not be doneby injecting the CRISPR DNA directlyinto a fully grown adult. Instead,scientists would take a sample of a persons bone marrow and alter the genes of those cells. Since bone marrow is responsible for producing red blood cells, the new altered bone marrow will produce cells with the new DNA.

Connelly saidthechangeswouldnot be instantaneous.The human body replaces a majority of its cells within 13 days, soit would take around two weeks for the newly edited gene to be present in the human body.

The ability to now alter genes of morecomplexorganisms brings with it a variety of applications. Plants can be changed to increase nutritional value and pesticidal properties,whereas bacteria can be used to generate hormones and medicines.

Dr. David Holtzman,an adjunct professor in the College of Science, understands how gene editing is used and what it could be used for.

Most people are familiar with it [gene editing] for things like modifying plants ...[but] there is a lot of misunderstanding about gene editing, Holtzman said.

There is a lot of misunderstanding about gene editing.

CRISPR has begun to work its way into at-home kits, where those with some scientificexpertise can genetically modify their own plants to glow or be a different color. This is fairly simple in the world of gene editing as it is changing a simple expressed trait one that isbiologically shown.

Genes decide what traits a person has, but that persons environment and what happens to their body determines how those traits are expressed. As gene editing becomes more and more innovative, Holtzman said that there are limitations to what gene editing can and cannot do.

It turns out most traits are more than one gene, Holtzman said.

Holtzman used hair color as an example. Numerous genes and sections of DNA code for what an individual's hair colorwill be. Itcan behard and time-consuming to find the right area of the DNA to target for modification.

Connelly talked about the idea of changing hair coloras well,but took it a few steps further. Shesuggestedthat we may start wanting to create offspring that all have blonde hair and blue eyes, which realistically we could accomplish. This then opens parents up to the ideas of having all male children or all female children.

In recent years, science has progressed faster than we could have thought.What appeared to be science fiction in the past is inching ever closer to our scientific reality.

The ability to do [new]things happens a lot faster than our understanding of what we are doing, Holtzman said.

Regardless of the potential scientific progress that could be made, Holtzman, Connelly and other members of the scientific community are having conversations about what should be done with this technology. Where should the limits lie, and how far should humans gowith genetic technology?

"Where should the limits lie, and how far should humansgowith genetic technology?"

If our parents changed our genes, they would also be changing the genes of all of our descendants by extension. Did they consent to something like that?

Some might argue, whether we gene edit or not, we dont really have control over what our parents did, Holtzman said. There is the possibility that if we changed [certain genes]then we can change them back.

Reversal isn't a guarantee, though.

Holtzman mentioned ways in which gene editing could greatly improve the quality of life for all humankind, such as curing Alzheimers disease. Connelly brought up how easy it would be to reduce the effects of aging using genetic modification.

The consequences of the choices made nowmay not affect the generation making them. As the movement to improve the genetic composition of the human race pushes forward,plots in sci-finovelsmay no longer be abstract, distant futures. Rather, for better or worse, they could bethe reality we are setting up for generations to come.

Read more:
Science Fiction Becoming Reality - Reporter Magazine

On the 12th of December, the United Kingdom will hold a general election. With the UKs exit from the European Union (Brexit) remaining unresolved, tensions are as high as ever. Once out of the EU, though, the UK could regain full control over its laws and regulations.

Though the election debate has centered around immigration, security and healthcare, the question of what direction the UK should take in terms of science policy persists. Will the UK manage to unleash the potential of its biotechnological sector and become a global advocate for innovation and consumer choice, or will it retain the EUs antiquated approach?

In a manifesto released in November, the Conservatives pledged to take the path of science-led, evidence-based policy to improve the quality of food, agriculture and land management. Previously, Prime Minister Boris Johnson promised to liberate the UKs biotech sector from the EUs anti-genetic modification rules.

The laws that concern genetically modified organisms in the UK are primarily based on European Union regulations. For years, the EU has backpedaled on agricultural innovation, preventing European consumers from accessing biologically enhanced food. This can be seen in the very limited number of genetically modified crops authorized for cultivation in the EU, and a very cumbersome and expensive process of importing genetically modified crops from other countries. In July 2018, the European Court of Justice (ECJ) decided that gene-edited plants should be regulated the same way that genetically modified organisms are regulated, rendering them practically illegal and hindering innovation even further.

If the UK chooses to move away from these EU-based regulations as a consequence of Brexit, it could become a forward-looking global biotech powerhouse.

The first step would be to replace fear-based skepticism of genetic modification with an evidence-based, pro-innovation approach. Despite popular rhetoric, there is no substantial scientific evidence behind the alleged health and environmental risks ascribed to GM products. Abandoning these baseless assertions and creating and sustaining the conditions under which UK farmers could innovate, lower their production costs, and use fewer chemicals would be an enterprising move on the part of the UK government.

Approving GM pest-resistant crops, for instance, could save about 60 million ($79 million) a year in pesticide use in the UK. Moreover, 60 million in savings would mean more leeway for competitive food pricing in a country where prices at the grocery store are rising 2 percent annually.

Once restrictive genetic modification laws are relaxed, it would be necessary to enable easy market access for GM foods. Under current EU legislation, products containing GMOs need to be labeled as such, and the requirements also apply to non-prepacked foods. It is legally established that such products (soy, for example) not only require written documentation but also should have an easily readable notice about their origin. No such rule exists with regards to foods that are 100% GMO-free, meaning there is explicit discrimination in place giving GMO-free food an unfair advantage on the market.

The EUs strict regulations on the use of GM technology have been, first and foremost, harmful to consumers, depriving them access to innovative options such as Impossible Foods plant-based burger, which so closely mimics meat thanks to an ingredient produced with the help of genetically engineered yeast. Vastly popular in the US and now expanding to Asia, vegan burgers using plant-based substitutes for meat and dairy products, are absent from the European market due to backwards-looking anti-GM rules.

The United Kingdom should strive for the smartest regulation in the field of approval and market access to GMOs. Relaxed regulations on gene-editing methods like CRISPR-Cas9 could also attract massive investment and lead to wide-reaching biotech innovation in the UK.

Enabling gene-editing is an essential part of unleashing scientific innovation in the United Kingdom after Brexit. Skepticism of gene-editing centers around the potential but largely exaggerated adverse effects of the technology and ignores the astonishing benefits that could accrue to both farmers and consumers.

If the UK manages to replace the EUs overly cautious biotech rules with a pro-innovation and prosperity-fostering regulatory scheme, it could become a true global biotech powerhouse. This is an ambitious, exciting, and above all, achievable future.

Maria Chaplia is a European Affairs Associate at the Consumer Choice Center. Visit her website and follow her on Twitter @mchapliaa

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Viewpoint: Conservatives say UK could break from 'outdated' EU GMO, CRISPR regulations if they sweep 'Brexit election' - Genetic Literacy Project

New York City, NY: Dec 12, 2019 Published via (Wired Release) The CRISPR Technology Market Report characterizes and briefs perusers about its items, applications, and particulars. The examination records key organizations working in the market and furthermore features the key changing course received by the organizations to keep up their quality. By utilizing SWOT investigation and Porters five power examination instruments, the qualities, shortcomings, openings, and malediction of key organizations are out and out referenced in the report. Each and every driving player in this worldwide market is profiled with subtleties, for example, item types, business outline, deals, fabricating base, candidate, applications, and particulars.

Key players inside the CRISPR Technology market are known through auxiliary investigation, and their pieces of the pie are resolved through essential and optional examination. All action shares split, and breakdowns are fearless exploitation auxiliary sources and checked essential sources. The CRISPR Technology Market report starts with a fundamental rundown of the exchange lifecycle, definitions, characterizations, applications, and exchange chain structure and each one these along can encourage driving players to see the extent of the Market, what attributes it offers and the manner in which itll satisfy clients needs.

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Major Players:

Thermo Fisher Scientific, Inc.Merck KGaAGenScript CorporationIntegrated DNA Technologies, Inc.Horizon Discovery GroupAgilent Technologies, Inc.Cellecta, Inc.GeneCopoeia, Inc.New England Biolabs, Inc.Origene Technologies, Inc.

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The research provides explanations to the accompanying key queries of CRISPR Technology industry:

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5. Which factors can avoid, challenge or even cutoff the improvement of the CRISPR Technology market the world over?

6. What are the odds or future conceivable outcomes for the business visionaries working in the industry for the measure time allotment, 2020 2029?

Table of Contents:

1. CRISPR Technology Market Survey.

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3. Global CRISPR Technology Market Race by Manufacturers.

4. Global CRISPR Technology Production Market Share by Regions.

5. Global CRISPR Technology Consumption by Regions.

6. Global CRISPR Technology Production, Revenue, Price Trend by Type.

7. Global CRISPR Technology Market Analysis by Applications.

8. CRISPR Technology Manufacturing Cost Examination.

9. Advertising Channel, Suppliers, and Clienteles.

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CRISPR Technology Market: 2020 With Top Competitors Analysis And Insights - Sound On Sound Fest

An effective andinnovative way to treat people with sickle cell anemia using gene therapy may soon be available thanks to efforts by several pharmaceutical companies, a Bloomberg report says.

Sickle cell anemia, a genetic defect that causes red blood cells to form in theshape ofa sickle, hinders the bodys ability to adequately distribute oxygen. This is due to atypical hemoglobin molecules, which is the protein in blood that transports oxygen. Sickle cell disease can be extremely painful, causing blood cells to get trapped in blood vessels and lead to heart failure, debilitating fatigue, strokes and blood clots.About 100,000 people suffer from sickle cell anemia in the U.S,with African Americansbeing disproportionately affected by this condition.

New developments with gene therapy, however, could work to have a positive impact on these symptoms. One of the innovative manufacturers, Bluebird Bio, stole the show at the annual conference of the American Society of Hematology in Florida. Its product, LentiGlobin, debuted positive results; in 17 patients treated with LentiGlobin,more than 40 percent of the hemoglobin in patients' red blood cells appearedin a healthier form thanks to gene therapy, per the article.

Bluebird isnt the only biotechnology making strides in gene therapies. Another potential treatment being researched is based on the technology called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), a gene-editing tool that is being used for a wide range of biomedical applications.

Documented in an NPR report, sickle cell patient Victoria Gray recently became the first person in the U.S. to have billions of her own cells genetically edited with CRISPR and reintroduced into her body. These cells will hopefully produce fetal hemoglobin to compensate for the faulty hemoglobin in Grays red blood cells. The trial is being expanded to include more patients and is being conducted by Vertex Pharmaceuticals and CRISPR Therapeutics of the Boston area.

Current treatments for sickle cell include blood and bone marrow transfusions and medication. Studies on gene therapy treatments have been encouraging so far, but there is more testing to be done before either CRISPR or LentiGlobin hits the market.

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Gene therapy could be a revolutionary new treatment for sickle cell disease - The Hill

BERKELEY, Calif.--(BUSINESS WIRE)--GenEdit, Inc., a developer of a novel polymer nanoparticle technology platform for non-viral- and non-lipid-based delivery of gene therapies, today announced that it has entered into a worldwide, exclusive license and collaboration agreement with Editas Medicine, Inc., a leading genome editing company. GenEdit has developed a comprehensive delivery system for CRISPR-based therapeutics, including gene knockout and gene repair therapies, to enable safer delivery options with improved efficiency.

"This license and collaboration agreement further validates the strength of our intellectual property portfolio and the potential of GenEdits technology," said Kunwoo Lee, Ph.D., co-founder and chief executive officer of GenEdit. "We are pleased to establish our relationship with Editas Medicine as they leverage our technology to develop potential genomic medicines."

Under the terms of the agreement, GenEdit has granted Editas Medicine an exclusive worldwide license, with rights to sublicense, to GenEdits Cpf1-based technologies. In return for these rights, GenEdit will receive undisclosed upfront and development milestone payments, including royalties on net sales of products incorporating the licensed intellectual property. In addition, GenEdit and Editas Medicine will collaborate on evaluating delivery of Cpf1-based technologies with GenEdits nanoparticle platform. Editas Medicine will provide research funding and have an option to continue development after the initial collaboration period.

GenEdits nanoparticle platform consists of a proprietary non-viral, non-lipid library of polymers that efficiently encapsulate and deliver cargo [RNA, DNA, protein and/or ribonucleic acid-protein complexes (RNP)] to specific tissues. The company screens the library to identify initial hits and then uses computational analysis and medicinal chemistry for iterative lead optimization. The company has used this platform to identify multiple candidate polymers for efficient and specific delivery of gene editing to a range of tissues.

"Compared to viral vectors and lipid-based nanoparticles, our approach has the potential for better targeting, more cargo, and lower manufacturing cost," said Timothy Fong, Ph.D., chief scientific officer of GenEdit. "In particular, our approach has the potential to enable in vivo gene editing of multiple tissues with CRISPR and expand the potential of gene therapies to treat more diverse sets of diseases."

About GenEdit

GenEdit was founded to transform the delivery of gene and gene editing therapies. We have synthesized the NanoGalaxy library of polymers that can encapsulate RNA, DNA, protein and/or RNP. Through advanced screening methods, computational analysis and iterative medicinal chemistry, we have demonstrated efficient delivery of gene editing cargo to specific tissues. We seek development partnerships for specific tissues and/or gene targets while advancing our internal pipeline of gene editing therapies.

For more information, please visit http://www.genedit.com.

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GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery - Business Wire

Its been just over a year since the dramatic announcement of the worlds first genome-edited babies using CRISPR technology. Since then, to the chagrin of some and the relief of others, there have been no more such announcements. This is due, in no small part, to discreet actions taken by the Peoples Republic of China, the World Health Organization (WHO) and the Russian Federation.

Read more: What is CRISPR gene editing, and how does it work?

In late November 2018, He Jiankui, a Chinese biophysicist, confirmed hed created genetically modified twins in an effort to provide the children with resistance to HIV. A few days later, he presented some of his work at the Second International Summit on Genome Editing in Hong Kong. At this meeting, He mentioned another ongoing pregnancy involving the use of a genetically modified embryo. To this day, we do not know the outcome of this pregnancy.

What we do know is that Chinas Ministry of Science and Technology condemned Hes actions and shortly thereafter, Chinas National Health Commission drafted new regulations on the clinical use of emerging biomedical technologies, including human genome editing. The final text of the Administrative Regulations for the Clinical Application of New Biomedical Technologies is not yet available and it is not known when these regulations will come into effect.

Based on the draft text open to public comment, research of the type conducted by He would require approval from Chinas highest administrative authority.

In the wake of Hes controversial experiment, the WHO convened a multi-disciplinary Expert Advisory Committee on Human Genome Editing to examine the scientific, ethical, social and legal challenges associated with human genome editing (both somatic and germ cell).

Specifically, the committee was tasked by the director general, Tedros Adhanom Ghebreyesus, to advise and make recommendations on appropriate governance mechanisms. The committee (of which I am a member) met for the first time in March 2019.

In June 2019, Russian molecular biologist Denis Rebrikov announced his plans to follow in Hes footsteps. Rebrikov would genetically modify early-stage human embryos in his lab and use those embryos to initiate a pregnancy that hopefully would result in the birth of healthy HIV-resistant offspring. Unlike He, however, Rebrikov planned to involve HIV-infected women in his research in an effort to address the risk of transmission of the virus in utero from the pregnant woman to her fetus. (Hes research involved HIV infected men.)

In response, on advice from the WHO Expert Advisory Committee, the WHO director general issued a statement calling on regulatory and ethics authorities in all countries to refrain from approving research on heritable human genome editing until its ethical and social implications had been properly considered.

Read more: Opening Pandora's Box: Gene editing and its consequences

Undeterred by the WHO announcement, in September and October 2019 Rebrikov, confirmed his intention to apply for permission to proceed with heritable human genome editing, but with a different focus. Though it was initially reported that Rebrikov felt a sense of urgency to help women with HIV, he was unable to find HIV-positive women who did not respond to standard anti-HIV drugs and who wanted to get pregnant to participate in his research.

So, instead of modifying the CCR5 gene which would provide future offspring with resistance to HIV, Rebrikov planned to modify the GJB2 gene to correct a mutation that causes a type of hereditary deafness. According to Rebrikov, there were several couples interested in participating in this research.

Meanwhile, the Russian government issued a statement making it clear that Rebrikov would not get regulatory approval for the proposed research.

In October 2019, the Ministry of Health of the Russian Federation affirmed that the use of heritable genome editing was premature. Further, the ministry officially endorsed the WHO position that it would be irresponsible and unacceptable to use genome-edited embryos to initiate human pregnancies.

Finally and most importantly the Ministry of Health explicitly stated that the WHO position, supported by the Russian Federation, should be decisive in the formation of country policies in this area.

This strong statement by the Ministry of Health of the Russian Federation is reassuring. It sets an important example for regulatory authorities around the world who support the WHOs efforts to develop effective governance instruments to deter and prevent irresponsible and unacceptable uses of genome editing of embryos to initiate human pregnancies.

In the last lines of my new book Altered Inheritance: CRISPR and the Ethics of Human Genome Editing I write:

As a direct consequence of increasingly audacious moves by some scientists to engineer future generations, important decisions must now be made decisions that will set a new course for science, society, and humanity. May these decisions be inclusive and consensual. May they be characterized by wisdom and benevolence. And, may we never lose sight of our responsibilities to us all.

Collectively, all of us (experts and non-experts) have a responsibility to make the best use of emerging technologies to improve the health and well-being of all people everywhere. This can only be achieved through collaborative effort on a global scale.

We need time to carefully consider the kind of world we want to live in and how human genome editing technology might or might not help us build that world. We cant do this work properly if scientists brashly go about the business of making genome-edited babies.

[ Youre smart and curious about the world. So are The Conversations authors and editors. You can read us daily by subscribing to our newsletter. ]

Read more here:
A year after the first CRISPR babies, stricter regulations are now in place - The Conversation CA

The Global CRISPR Genome Editing Market report study includes an elaborative summary of the CRISPR Genome Editing market that provides in-depth knowledge of various different segmentations. CRISPR Genome Editing Market Research Report presents a detailed analysis based on the thorough research of the overall market, particularly on questions that border on the market size, growth scenario, potential opportunities, operation landscape, trend analysis, and competitive analysis of CRISPR Genome Editing Market. The information includes the company profile, annual turnover, the types of products and services they provide, income generation, which provide direction to businesses to take important steps. CRISPR Genome Editing delivers pin point analysis of varying competition dynamics and keeps ahead of CRISPR Genome Editing competitors such as Editas Medicine, CRISPR Therapeutics, Horizon Discovery, Sigma-Aldrich, Genscript, Sangamo Biosciences, Lonza Group, Integrated DNA Technologies, New England Biolabs, Origene Technologies, Transposagen Biopharmaceuticals, Thermo Fisher Scientific, Caribou Biosciences, Precision Biosciences, Cellectis, Intellia Therapeutics.

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The main objective of the CRISPR Genome Editing report is to guide the user to understand the CRISPR Genome Editing market in terms of its definition, classification, CRISPR Genome Editing market potential, latest trends, and the challenges that the CRISPR Genome Editing market is facing. In-depth researches and CRISPR Genome Editing studies were done while preparing the CRISPR Genome Editing report. The CRISPR Genome Editing readers will find this report very beneficial in understanding the CRISPR Genome Editing market in detailed. The aspects and information are represented in the CRISPR Genome Editing report using figures, bar-graphs, pie diagrams, and other visual representations. This intensifies the CRISPR Genome Editing pictorial representation and also helps in getting the CRISPR Genome Editing industry facts much better.

.This research report consists of the worlds crucial region market share, size (volume), trends including the product profit, price, Value, production, capacity, capability utilization, supply, and demand and industry growth rate.

Geographically this report covers all the major manufacturers from India, China, the USA, the UK, and Japan. The present, past and forecast overview of the CRISPR Genome Editing market is represented in this report.

The Study is segmented by following Product Type, Genetic Engineering, Gene Library, Human Stem Cells, Others

Major applications/end-users industry are as follows Biotechnology Companies, Pharmaceutical Companies, Others

CRISPR Genome Editing Market Report Highlights:

1)The report provides a detailed analysis of current and future market trends to identify the investment opportunities2) In-depth company profiles of key players and upcoming prominent players3) Global CRISPR Genome Editing Market Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, and recommendations)4) Strategic recommendations in key business segments based on the market estimations5) To get the research methodologies those are being collected by CRISPR Genome Editing driving individual organizations.

Research Parameter/ Research Methodology

Primary Research:

The primary sources involve the industry experts from the Global CRISPR Genome Editing industry including the management organizations, processing organizations, analytics service providers of the industrys value chain. All primary sources were interviewed to gather and authenticate qualitative & quantitative information and determine future prospects.

In the extensive primary research process undertaken for this study, the primary sources industry experts such as CEOs, vice presidents, marketing director, technology & innovation directors, founders and related key executives from various key companies and organizations in the Global CRISPR Genome Editing in the industry have been interviewed to obtain and verify both qualitative and quantitative aspects of this research study.

Secondary Research:

In Secondary research crucial information about the industry value chain, the total pool of key players, and application areas. It also assisted in market segmentation according to industry trends to the bottom-most level, geographical markets and key developments from both market and technology oriented perspectives.

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Global CRISPR Genome Editing Market 2019 by Company, Regions, Type and Application, Forecast to 2025 - The Market-News 24

University of Otago researchers have discovered how viruses that specifically kill bacteria can outwit bacteria by hiding from their defenses, findings which are important for the development of new antimicrobials based on viruses and provide a significant advance in biological knowledge.

Lead researcher Professor Peter Fineran explains that the rise in multi-drug resistant bacteria is leading to the development of alternative therapeutics, including viruses that specifically kill bacteria, called bacteriophages, often referred to as phages. However, bacteria can become resistant to phages.

This is professor Peter Fineran. Credit: University of Otago

Phages are the most abundant biological entities on the planet and are important for global ecosystems, but they can also be used to kill bacterial pathogens. To defend themselves from the phage invasion, bacteria have developed CRISPR-Cas defense systems immune systems within the bacteria. But the phages have come up with many ways to avoid these bacterial defenses.

In the study published December 9, 2019, in Nature Microbiology, the team at the University of Otago discovered a widespread method used by phages to hide from bacterial defenses. They discovered a jumbo phage which, as the name suggests, is very big, with hundreds of genes. This phage is not recognized by CRISPR-Cas defenses that would normally cut up the genetic DNA instructions to make many new phages.

Ph.D. student in the Department of Microbiology and Immunology and first author of the study, Lucia Malone says it made the researchers question how this phage escapes recognition.

We had molecular and genetic evidence for what was happening, but we really needed to see directly inside these tiny bacteria, which if 100 lined up side-by-side would be the width of a human hair, Ms. Malone says.

This is first author of the study, PhD student Lucia Malone. Credit: University of Otago

This was made possible using a new spinning disk confocal microscope for high-resolution imaging of live cells the only one with this capability in New Zealand that was recently set up by Dr. Laura Gumy, a new group leader at the University of Otago.

When phages infected the bacteria, we could see their DNA was encased by a physical shield and hidden from the CRISPR-Cas defence systems that couldnt gain access, Dr. Gumy explains.

However, bacteria have another trick up their sleeve. To take over the host, the phages must produce RNA messages that leave this protective compartment. This is the Achilles heel of these phages and can be destroyed by a special group of CRISPR-Cas defenses that recognize RNA messages, Ms Malone says.

Dr. Fineran explains the study broadens the knowledge of intricate phage-host interactions and demonstrates that jumbo phages are less susceptible to bacterial defense systems than some other phages.

From a biological perspective, our results provide exciting new insights into how phages evade bacterial defense systems.

This is important because the rise of the multi-drug resistant bacteria is an issue of global concern, which has led to a renewed interest in using phages as anti-bacterials and jumbo phages may provide excellent therapeutics.

Reference: A jumbo phage that forms a nucleus-like structure evades CRISPRCas DNA targeting but is vulnerable to type III RNA-based immunity by Lucia M. Malone, Suzanne L. Warring, Simon A. Jackson, Carolin Warnecke, Paul P. Gardner, Laura F. Gumy and Peter C. Fineran, 9 December 2019, Nature Microbiology.DOI: 10.1038/s41564-019-0612-5

Link:
New Viral Strategy to Escape Detection Discovered by Researchers - SciTechDaily

Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market report offers detailed analysis and a five-year forecast for the global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology industry. Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology market report delivers the insights which will shape your strategic planning as you estimate geographic, product or service expansion within the Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology industry.. Global Clustered Regularly Interspaced Short Palindromic Repeats(CRISPR) Technology Market Report is a professional and comprehensive research report on the worlds major regional market conditions, focusing on the main regions (North America, Europe and Asia-Pacific) and the main countries (United States, Germany, United Kingdom, Japan, South Korea and China).

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More than a year ago, the world was shocked by Chinese biophysicist He Jiankui's attempt to use CRISPR technology to modify human embryos and make them resistant to HIV, which led to the birth of twins Lulu and Nana.

Now, crucial details have been revealed in a recent release of excerpts from the study, which have triggered a series of concerns about how Lulu and Nana's genome was modified.

CRISPR is a technique that allows scientists to make precise edits to any DNA by altering its sequence.

When using CRISPR, you may be trying to "knock out" a gene by rendering it inactive, or trying to achieve specific modifications, such as introducing or removing a desired piece of DNA.

Gene editing with the CRISPR system relies on an association of two proteins. One of the proteins, called Cas9, is responsible for "cutting" the DNA. The other protein is a short RNA (ribonucleic acid) molecule which works as a "guide" that brings Cas9 to the position where it is supposed to cut.

The system also needs help from the cells being edited. DNA damage is frequent, so cells regularly have to repair the DNA lesions. The associated repair mechanisms are what introduce the deletions, insertions or modifications when performing gene editing.

Jiankui and his colleagues were targeting a gene called CCR5, which is necessary for the HIV virus to enter into white blood cells (lymphocytes) and infect our body.

One variant of CCR5, called CCR5 32, is missing a particular string of 32 "letters" of DNA code. This variant naturally occurs in the human population, and results in a high level of resistance to the most common type of HIV virus.

Jankui's team wanted to recreate this mutation using CRISPR on human embryos, in a bid to render them resistant to HIV infection. But this did not go as planned, and there are several ways they may have failed.

First, despite claiming in the abstract of their unpublished article that they reproduced the human CCR5 mutation, in reality the team tried to modify CCR5 close to the 32 mutation.

As a result, they generated different mutations, of which the effects are unknown. It may or may not confer HIV resistance, and may or may not have other consequences.

Worryingly, they did not test any of this, and went ahead with implanting the embryos. This is unjustifiable.

A second source of errors could have been that the editing was not perfectly efficient. This means that not all cells in the embryos were necessarily edited.

When an organism has a mixture of edited and unedited cells, it is called a "mosaic". While the available data are still limited, it seems that both Lulu and Nana are mosaic.

This makes it even less likely that the gene-edited babies would be resistant to HIV infection. The risk of mosaicism should have been another reason not to implant the embryos.

Moreover, editing can have unintended impacts elsewhere in the genome.

When designing a CRISPR experiment, you choose the "guide" RNA so that its sequence is unique to the gene you are targeting. However, "off-target" cuts can still happen elsewhere in the genome, at places that have a similar sequence.

Jiankui and his team tested cells from the edited embryos, and reported only one off-target modification. However, that testing required sampling the cells, which were therefore no longer part of the embryos - which continued developing.

Thus, the remaining cells in the embryos had not been tested, and may have had different off-target modifications.

This is not the team's fault, as there will always be limitations in detecting off-target and mosaicism, and we can only get a partial picture.

However, that partial picture should have made them pause.

Above, we have described several risks associated with the modifications made on the embryos, which could be passed on to future generations.

Embryo editing is only ethically justifiable in cases where the benefits clearly outweigh the risks.

Technical issues aside, Jiankui's team did not even address an unmet medical need.

While the twins' father was HIV-positive, there is already a well-established way to prevent an HIV-positive father from infecting embryos. This "sperm washing" method was actually used by the team.

The only benefit of the attempted gene modification, if proven, would have been a reduced risk of HIV infection for the twins later in life.

But there are safer existing ways to control the risk of infection, such as condoms and mandatory testing of blood donations.

Gene editing has endless applications. It can be used to make plants such as the Cavendish banana more resistant to devastating diseases. It can play an important role in the adaptation to climate change.

In health, we are already seeing promising results with the editing of somatic cells (that is, non-heritable modifications of the patient's own cells) in beta thalassemia and sickle cell disease.

However, we are just not ready for human embryo editing. Our techniques are not mature enough, and no case has been made for a widespread need that other techniques, such as preimplantation genetic testing, could not address.

There is also much work still needed on governance. There have been individual calls for a moratorium on embryo editing, and expert panels from the World Health Organisation to UNESCO.

Yet, no consensus has emerged.

It is important these discussions move in unison to a second phase, where other stakeholders, such as patient groups, are more broadly consulted (and informed). Engagement with the public is also crucial.

Dimitri Perrin, Senior Lecturer, Queensland University of Technology and Gaetan Burgio, Geneticist and Group Leader, The John Curtin School of Medical Research, Australian National University.

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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New Details About The Infamous 'CRISPR Babies' Experiment Have Just Been Revealed - ScienceAlert

We live in a time when science and technology are having an impact on our society in more and more ways. And the decisions that shape how these new fields of knowledge develop ultimately affect all of us.

When I studied biology in high school, I didnt learn about DNA for a very simple reason. The work of Francis Crick, James Watson, Rosalind Franklin and others who unlocked the structure of the basic code of life was still years away. The idea of engineering human beings? Well, that was firmly the stuff of science fiction, like Aldous Huxleys dystopian novel Brave New World (published a year after my birth). It seemed as likely as, say, going to the moon.

There are a few inferences you can make from this framing of my life. One is that I have been on the planet for a while. The other is the speed of change in what we know about what life is, and how we can control it, has accelerated at a rapid rate. Now we as a species are on the precipice of being able to manipulate the very building blocks of human evolution, not to mention wield unpredictable change on the greater world around us. Even as I commit that thought to paper, I pause in awe at its implications.

I have lived through eventful times and my job as a journalist has been to chronicle wars, presidents and sweeping social movements such as civil rights. I have seen a world in flux, but when I try to peer into the future I come to the conclusion that this story of humankinds ability to understand life on its most intimate level and be able to tinker with it for our benefit or detriment is likely to be the biggest one I will ever cover.

We are living in one of the greatest epochs of human exploration and it will shape our world as profoundly as the age of the transoceanic explorers. It is just that the beachheads on which we are landing and the continents we are mapping comprise a world far too small to see with the naked eye. Some of it is even invisible to our most powerful microscopes.

This brings me to a term that has become a big part of my life over the last few years: Crispr. Perhaps you know of it. Perhaps you dont. When I first heard of it, I thought it might be a new brand of toaster. I now know its an extremely powerful tool for editing genes in seemingly any organism on Earth, including humans. Scientists doing basic research have been uncovering the mechanisms of life for decades. They have been creating tools for modifying individual genes but Crispr is one of those revolutions where what researchers thought might be possible in the distant horizon is suddenly available now. Its cheap, its relatively simple and its remarkably precise.

I immediately knew that this was a story that needed telling. Human Nature, the resulting film full disclosure, I am executive producer came out of our conversations with scientists. They tend not to be the type of people who hype things but when they talk about Crispr you can feel the urgency in their voices. This is something you need to know about. All of you. If you are worried about your health or the health of your children. If you are concerned about how we might need to engineer our planet in the face of the climate crisis. If you are in finance, law or the world of tech. This will shape all of it.

And as we grapple with the unintended consequences of the internet and social media, as we try to make progress against a heating planet, I humbly submit that we as a species tend not to be good at thinking through where we are going until a crisis is already upon us. I fervently hope with Crispr that we can start the conversation sooner. That we can start it now. Thats why we made the film.

To be clear, we are probably a long way from designing babies to be more intelligent or more musically inclined. Life is just too complex for that, at least right now. More immediately, there is so much about this technology that is very exciting. As someone who remembers a time when my classmates were struck down with childhood diseases for which we now have vaccines, I know science can have profound applications for human health. Crispr could cure genetic diseases such as sickle cell and Huntingtons. It is being tested against cancers and HIV. It could also potentially be used to make crops more drought-resistant or food more nutritious.

On the other hand, we are walking closer to a world Aldous Huxley foresaw. What does it mean to be human? Where should we draw the boundaries beyond which we dare not cross? The inspiring researchers we talked to for the film know that the ethical and moral questions this technology raises are not for them to decide. Science has given us the tools, but not the answers. This is up to us, all of us. We need to be informed. We need to be honest with whats real and whats not. And we need to add our voices to a global conversation. Thats part of our responsibility as humans living on Earth today.

Dan Rather is one of the USs most feted journalists. He anchored CBS Evening News for 24 years

Human Nature is in UK cinemas now before a university town tour in the new year, wondercollaborative.org/human-nature-documentary-film/#screenings . It will be shown on BBC Storyville in spring/summer 2020

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Gene editing will let us control our very evolution. Will we use it wisely? - The Guardian

Bengaluru: What must it have felt like to be a cotton spinner or an iron maker in England in the 1820s in the midst of an industrial revolution? Exactly 200 years later, we may be on the verge of another era of momentous change: the internet revolution. With internet access expanding dramatically post the early 1990s, a slew of new technologies have now matured to a point where fundamental change constantly seems to be right around the corner.

On the doorstep of a brand new decadethe 2020swhat new frontiers may Artificial Intelligence (AI) or gene editing open up? Will we soon have robot bosses? Will mixed reality change the way we consume entertainment and sports? Will we be able to cure 90% of all genetic diseases by the end of the decade? We take a look at five technologies that could alter India and the world. This may not be a definitive or even exhaustive list, but it is a list of things that could change the way we live, work, and play sooner than we think.

Mixed reality

Imagine watching a football match, not on your TV but on a virtual reality (VR) headset that streams the match live and projects interesting stats on the fly with the help of augmented reality (AR). Mumbai-based VR startup Tesseract, now owned by Mukesh Ambanis Reliance Jio, is promising a future like that with its Quark camera, Holoboard headset, and the high internet speeds of Jio Fiber. Similarly, a Hyderabad-based mixed reality startup called Imaginate enables cross-device communication over VR and AR wearables for better enterprise collaboration in the industrial sector.

Despite the much-hyped yet unmet expectations from the likes of Google Glass, Microsoft HoloLens and Facebooks Oculus, Tesseract and Imaginate simply underscore how the fusion of AR and VR technologies the combination of which is popularly known as Mixed Reality or MR is coming of age and is no longer in the realm of just sci-fi movies like Blade Runner 2049, where Officer K played by Ryan Gosling develops a relationship with his artificial intelligence (AI) hologram companion Joi.

For instance, AI-powered chatbots today can not only conduct a conversation in natural language via audio or text but they can be made more powerful with a dose of mixed reality. Last May, Fidelity Investments created a prototype VR financial advisor named Cora to answer client queries using a suite of tools from Amazon Web Services. Researchers in Southampton have built a device that displays 3D animated objects that can talk and interact with onlookers.

The Chinese government-run Xinhua News Agency has the worlds first AI-powered news anchor, whose voice has been modelled to resemble a real human anchor working for the agency. Going a step further, Japan-headquartered DataGrid Inc. uses generative adversarial networks (GANs) to develop its so-called whole body model automatic generation AI" that automatically generates full-length images of non-existent people with high resolutions.

Nevertheless, challenges abound when dealing MR-and AI-powered robots, humanoids, and human avatars. For one, whenever a company generates human bodies and faces, concerns over deep fakes and cheap fakes will always rear their heads. Second, data collection will continually raise concerns over security and privacy. Third, theres always the concern regarding the fairness of an AI algorithm when it is deployed to do human tasks like giving financial advice. Last, but not the least, theres also the question of whether AI bots should be allowed to pose as humans. This will continually pose a challenge and opportunity for technologists and policy makers.

Future of solar

Heliogen, a company that has billionaire philanthropist Bill Gates as one of its investors, says it has created the worlds first technology that can commercially replace fuels with carbon-free, ultra-high temperature heat from the sun. With its patented technology, Heliogens field of mirrors acts as a multi-acre magnifying glass to concentrate and capture sunlight.

This is just a case in point that solar technologies have evolved a lot since they first made their debut in the 1960s. For instance, solar roadwayspanels lining the surface of highwayshave already popped up in the Netherlands. Floating solar, on its part, is providing a credible option to address land use concerns associated with wide scale solar implementations. A French firm called Ciel et Terre, for instance, has projects set up in France, Japan, and England. Other parts of the world, including India and California in the US, are piloting similar floating solar initiatives.

Space-based solar technology is another exciting arena. India, China and Japan are investing heavily in these technologies right now. The Japan Aerospace Exploration Agencys (JAXA) Space Solar Power Systems (SSPS) aims to transmit energy from orbiting solar panels by 2030. Further, researchers at the VTT Technical Research Centre in Finland have used solar and 3D printing technologies to develop prototypes of what they have christened as energy harvesting trees".

With solar power cheaper than coal in most countries in the world, its worth scaling up these technologies.

Indians and robot bosses

Between 400 and 800 million individuals around the world could be displaced by automation and would need to find new jobs by 2030, predicted a December 2017 survey by consultancy firm McKinsey. The Future of Jobs 2018 report by the World Economic Forum (WEF) suggests that 75 million jobs may be lost to automation by 2022, but adds that another 133 million additional new roles will be created.

Given that many of the automated jobs are being taken away by AI-powered chatbots and intelligent robots, would humans eventually have to work for a robo boss? This, however, may not be as big a concern as it is made out to be. According to the second annual AI at Work study conducted by Oracle and Future Workplace, people trust robots more than their managers. The study, released this October, notes that workers in China (77%) and India (78%) have adopted AI over 2X more than those in France (32%) and Japan (29%). Further, workers in India (60%) and China (56%) are the most excited about AI, while men have a more positive view of AI at work than women.

Oracle and Future Workplace also found that 82% of the workers believe robot managers are better at certain tasks, such as maintaining work schedules and providing unbiased information, than their human counterparts. And almost two-thirds (64%) of workers worldwide say they would trust a robot more than their human manager. In China and India, that figure rises to almost 90%.

On the other hand, the respondents felt managers can outdo robots when it comes to understanding their feelings, coaching them, and creating a healthy work culture. Whether humans eventually serve a robo boss or not remains to be seen. However, we can be certain of one thing: in the near future, we will increasingly see humans collaborating with smart robots.

Future of payments

Everyone can be a merchant, and every device can be an acceptance device," Accenture noted in its 2017 Driving the Future of Payments report. This trend has only accelerated over the last two years, especially with banks coming to terms with the fact that young customers, especially those living in urban areas, prefer net banking and mobile banking and would seldom, or never, want to visit a bank branch if offered that choice.

Bitcoin and cryptocurrency investors, for instance, have not lost faith in this disruptive currency despite the run with volatility, and despite the industry being viewed with a lot of suspicion by most governments around the world, including India. Fintechs too, with their innovative technology solutions like AI-powered bots and contactless payments to name a few, have only made the payments ecosystem more inclusive, disruptive, and challenging. In India, especially, the governments Aadhaar-enabled payments system and the Unified Payments Interface (UPI) have revolutionized the payments ecosystem. The total volume of UPI transactions in the third quarter of calendar 2019 touched 2.7 billiona 183% rise over the same July-September quarter a year ago. In terms of value, UPI clocked 4.6 trillionup 189% over the same period a year ago, according to the Worldlines India Digital Payments Report-Q3 2019.

However, the number of transactions done on mobile wallets was 1.04 billiononly a 5% rise over the previous year period.

QR codes, according to the report, will continue to be used for payments, and the internet of things (IoT) is set to dominate micro payments by transforming connected devices into payment channels, though the pace of adoption of 5G by countries like India will be the key.

Nevertheless, cash that has been in existence for over 3000 years in different forms is not going to disappear in a hurry. Trust and security will continue to remain the operative words in digital payments.

Making sense of gene editing

When Dolly the sheep made news for becoming the first mammal ever to be cloned from another individuals body cell, many expected human cloning to follow soon. Dolly died over 16 years ago, and subsequently animals, including monkeys and dogs, continue to be cloned successfully. Yet, no human being has yet been cloned in real life.

While human cloning, which may or may not eventually happen, is bound to raise a lot of alarm bells given the moral implications surrounding the issue, the fact is that human genomes, or genes, are being routinely edited in a bid to find solutions for what are today considered to be incurable genetically inherited diseases.

Researchers are using a gene editing tool known as CRISPR-Cas9. CRISPR, which stands for Clusters of Regularly Interspaced Short Palindromic Repeats, is a tool that allows researchers to easily alter DNA sequences and modify gene function. The protein Cas9 (CRISPR-associated, or Cas) is an enzyme that acts like a pair of molecular scissors capable of cutting strands of DNA.

CRISPR-Cas9 is primarily known for its use in treating diseases like AIDS, amyotrophic lateral sclerosis (ALS), and Huntingtons disease. Two patients, one with beta thalassemia and one with sickle cell disease, have potentially been cured of their diseases, reveal results from clinical trials that were jointly conducted by Vertex Pharmaceuticals and CRISPR Therapeutics. The results released this November involved using Crispr to edit the genes of these patients.

Researchers are now looking to extend its use to tackle famine, lend a hand in creating antibiotics, and even wipe out an entire species such as malaria-spreading mosquitoes. Further, by genetically engineering a persons bone marrow cells, researchers can reprogram their immune and circulatory systems. Some new cancer treatments are based on this. Moreover, looking at the DNA of the collection of microbes in your gut can help with digestive disorders, weight loss, and even help understand mood changes.

Closer home, scientists at the Institute of Genomics and Integrative Biology (IGIB) and the Indian Institute of Chemical Biology (CSIR-IICB) are trying to correct genetic mutations in their laboratories using CRISPR Cas9 with encouraging preliminary results. But due to regulatory and ethical concerns, it may take a while before they can use this on humans.

IGIB also sells CRISPR products such as Cas9 proteins and its variants to educational institutes at reduced prices in a bid to encourage use of the technology.

The US Food and Drug Administration (FDA), on its part, considers any use of CRISPR-Cas9 gene editing in humans to be gene therapy and rules that the sale of DIY kits to produce gene therapies for self-administration is illegal. India, too, has banned the use of stem cell therapy for commercial use following concerns over rampant malpractice".

CRISPR-Cas9, thus, remains a work in progress and countries should have policies to govern its use. Meanwhile, one can watch out for an upgrade to CRISPR called Prime, which theoretically has the ability to snip out more than 90% of all genetic diseases.

Read more:
Five technologies that may alter India in 2020 - Livemint

In the middle of November, Editas Medicine (NASDAQ:EDIT) and Celgene (NASDAQ:CELG) announced changes to a development pact originally formed in 2015 with Juno Therapeutics, which is now part of Celgene. The agreement was amended in 2018, too, so the fact that changes were made wasn't necessarily big news. Editas received a $70 million upfront payment for executing the amended agreement, which was interpreted as the main takeaway from the announcement.

The announcement barely registered with investors and few gave it much thought for too long, especially after promising early results from the first clinical trials using a CRISPR-based medicine were announced by CRISPR Therapeutics days later.

But revisiting the amended collaboration agreement, and specifically what changes were made, hints at the long-term development plans of Editas Medicine. In short, it now has full control over an important class of immune cells. Whether that means the gene-editing pioneer lands another major development partner or goes full-steam ahead alone, investors can't overlook the significance.

Image source: Getty Images.

The basic scientific goal of the collaboration hasn't changed. Editas Medicine will use its gene-editing technology platform to engineer T cell receptors (TCR), while Juno Therapeutics will leverage its immunotherapy leadership to develop the engineered cellular medicines in clinical trials.

Why engineer TCRs? Immune cells rely on their receptors to identify targets, such as pathogenic bacteria and cancer cells. But immune cell receptors can be confused by molecules secreted within the tumor microenvironment, forcing them to halt their attack. They can also incorrectly attack an individual's own cells to trigger an autoimmune disease. A more recent concern stems from cellular medicines derived from a donor. Since the donor cells present different receptors compared to what the recipient's native T cells carry, the recipient's immune system (correctly) identifies the immunotherapy as a foreign substance, attacks it, and renders it less effective and less safe.

Therefore, it makes sense to engineer TCRs to create more potent and stealthier immunotherapies that are less likely to be tricked. Editas Medicine and Celgene still intend to do just that, albeit with subtle, yet important, differences to their development agreement.

Consideration

Previous Agreement (2015, 2018)

Amended Agreement (2019)

Focus

Cancer

Cancer and autoimmune diseases

Types of cells

CAR-T cells, alpha-beta T cells, gamma-delta T cells

Alpha-beta T cells

Juno Therapeutics exclusivity

Editas Medicine prohibited from all other work with CAR-T and TCRs in oncology

Editas Medicine prohibited from all other work on alpha-beta T cells and T cells derived from pluripotent stem cells

Upfront payment

$57.7 million (includes milestones collected under agreement)

$70 million

Milestone potential

$920 million plus tiered royalties

$195 million plus tiered royalties

Data source: SEC filings.

Essentially, Editas Medicine and Celgene have scaled back their original agreement in cancer and expanded their work to include autoimmune diseases. The most important detail is that the amended agreement allows the gene-editing pioneer to pursue the development of gamma-delta T cells, which were previously under the exclusive control of Juno Therapeutics. What does that mean?

Image source: Getty Images.

Without getting too far into the weeds, there are two main types of TCRs: alpha-beta and gamma-delta. The name refers to the molecular structure of the receptor, but that's not the important part.

Gamma-delta T cells, which comprise only about 5% of the T cells in your body, are thought to be one of the missing links in our understanding of the immune system. They're a mysterious bunch, but there could be significant value residing in the knowledge gaps.

These unique immune cells are governed by their own unique set of rules (relative to their alpha-beta peers) and straddle the innate immune system (what we're programmed with at birth) and adaptive immune systems (what's programmed as we encounter new environments throughout life). Gamma-delta T cells could be tinkered with in gut microbiome applications, to treat cardiovascular diseases, and to neutralize antibiotic-resistant infections. But the nearest commercial target of the mysterious immune cells is likely to be treating solid tumor cancers.

They possess potent anti-tumor activity where current immunotherapies fail, such as attacking cancer cells that lack tumor-specific antigens to target or that have become immune to checkpoint inhibitors. In fact, there's a link between certain cancer outcomes and the activity of specific gamma-delta T cells.

Given that, why would Celgene amend the agreement to ditch the rare subset of immune cells? Well, in August 2019, Celgene inked with a start-up called Immatics to develop engineered TCRs. The start-up's platform is based on gamma-delta tech.

Don't feel too bad for Editas Medicine, though. SEC filings reveal that the gene-editing pioneer didn't receive any money from the original collaboration deal with Celgene in the first nine months of 2019. That suggests the work had stalled or that the amendment was being hammered out for some time. The gene-editing pioneer wrestled back control of the tech and took a $70 million upfront payment to boot. While the potential milestone payments in the amended agreement are significantly lower than the originally promised bounty, Editas Medicine can offset that by signing a lucrative collaboration deal with a new partner.

There should be plenty of interest. Fellow gamma-delta T cell developer Adicet Bio recently landed an $80 million series B round funded in part by Johnson & Johnson, Regeneron,Samsung Biologics(not the same company as the electronics powerhouse), and Novartis. There's also Immatics, GammaDelta Therapeutics, and a handful of other start-ups making noise in the space.

Some competitors are directly engineering gamma-delta cells, and others are developing molecules to trigger the immune cells into action. Editas Medicine believes it has the edge, as it has a relatively precise and efficient method for engineering immune cells: gene editing.

The amended collaboration deal between Editas Medicine and Celgene received relatively little attention from investors. Perhaps that was a good thing, as Wall Street likely would have overreacted to the reduced scope of development and milestones. But investors that take the time to understand the details might be intrigued by the new research avenue for the gene-editing stock.

Can Editas Medicine become a leading force in gamma-delta T cell development? Perhaps. While it isn't the only company wielding a gene-editing platform, and CRISPR gene editing isn't the only type of gene editing, the company is well-positioned to take advantage of the opportunity. Investors will have to wait to see how (or if) the development strategy evolves around the new tech.

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Celgene Gave This Tech Back to Editas Medicine, but It Could Prove Valuable - The Motley Fool

One of the most popular methods for inserting therapeutic genes into cells to treat disease is to transport them using a virus that has been stripped of its infectious properties. But those noninfectious viruses can still sometimes touch off dangerous immune responses.

A team from Johns Hopkins Medicine is proposing an alternative method for transporting large therapies into cellsincluding genes and even the gene-editing system CRISPR. Its a nano-container made of a polymer that biodegrades once its inside the cell, unleashing the therapy. The researchers described the invention in the journal Science Advances.

The team, led by biomedical engineer Jordan Green, Ph.D., was inspired by viruses, which have many properties that make them ideal transport vehicles. They have both negative and positive charges, for example, which allows them to get close to cells. So Green and his colleagues developed a polymer containing four molecules with both positive and negative charges. They used it to make a container that interacts with the cell membrane and is eventually engulfed by it.

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The Hopkins researchers performed four experiments to prove the nanocontainers would travel into cells and deliver complex therapies once inside. First, they packaged a small protein into the polymer material and mixed it with mouse kidney cells in a lab dish. Using fluorescent tags, they confirmed that the protein made it into the cells. Then they repeated the experiment with a much larger medicinehuman immunoglobulinand observed that 90% of the kidney cells received the treatment.

From there, they made the payload even bulkier, packaging the nanocontainers with the gene-editing system CRISPR. With the help of fluorescent signals, they were able to confirm that CRISPR went to work once inside the cells, disabling a gene 77% of the time.

"That's pretty effective considering, with other gene-editing systems, you might get the correct gene-cutting result less than 10 percent of the time," said graduate student Yuan Rui in a statement.

Finally, the Hopkins researchers injected CRISPR components into mouse models of brain cancer using the polymer nanocontainers. Again they saw evidence that successful gene editing had occurred.

Developing improved methods for gene therapy is a priority in the field. In October, for example, scientists at Scripps Research described a way to use a small molecule called caraphenol A to lower levels of interferon-induced transmembrane (IFITM) proteins, which could, in turn, allow viral vectors to pass more easily into cells. And earlier this year, an Italian team described a method for including the protein CD47 in lentiviral vectors to improve the transferring of therapeutic genes into liver cells.

The next step for Hopkins researchers Rui and Green is to improve the stability of the nanocontainers so they can be injected into the bloodstream. They hope to be able to target them to cells that have certain genetic markers, they reported.

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Hopkins team invents non-viral system for getting gene therapy into cells - FierceBiotech

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WASHINGTON DC, United States (AFP) In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old told AFP in an e-mail.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as molecular scissors.

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

It's all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the co-founder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not such as making normal red blood cells, in Victoria's case, or making tumour-killing super white blood cells for a cancer patient.

Crispr goes even further; instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, that's the time it had to take, he told AFP from Paris.

It took a generation for gene therapy to become a reality. Now, it's only going to go faster.

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to US$2 million meaning patients face gruelling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

It's very easy to do if you don't care about the consequences, Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

I'm a bacteriologist we've been talking about bioterrorism for years, she said. Nothing has ever happened.

Now you can read the Jamaica Observer ePaper anytime, anywhere. The Jamaica Observer ePaper is available to you at home or at work, and is the same edition as the printed copy available at http://bit.ly/epaperlive

Originally posted here:
2019: The year gene therapy came of age - Jamaica Observer

In its latest trading session, CRISPR Therapeutics AG (NASDAQ:CRSP) spiked up by 2.3% here is what that looked like (as of 2019-12-09):

Whats the reason for these moves? To understand this better, it is helpful to analyze some technical indicators. As we see it, you should pay the most attention to the following:

A Money Flow Index (MFI) of 100.0, a Relative Strength Index (RSI) of 50.0, a True Strength Index (TSI) of 100.0, a Moving Average Convergence Divergence (MACD) of -0.016, an Average Directional Movement Index (ADX) of unknown, an Average True Range (ATR) of 1.429, an Accumulation/Distribution Index (ADI) of 1.429, an On-Balance Volume (OBV) of 557.672, and a Chaiikin Money Flow (CMF) of -70.71. 2.492

Now lets plug these indicators in. Consulting Trading View, we can conclude the following:

That is what an analysis of technical indicators seems to indicate for CRISPR Therapeutics AG (NASDAQ:CRSP). Note this is technical analysis only! You should do fundamental research as well, and do not just rely on this we take no responsibility for any losses incurred if you buy or sell based on the above.

More here:
CRISPR Therapeutics AG (NASDAQ:CRSP) Spiked Up 2.3% Here's Why - Lateral Line

Wells Fargo analyst Jim Birchenough maintained a Buy rating on Crispr Therapeutics AG (CRSP Research Report) yesterday. The companys shares closed last Monday at $72.37, close to its 52-week high of $74.00.

According to TipRanks.com, Birchenough is a 5-star analyst with an average return of 21.4% and a 51.0% success rate. Birchenough covers the Healthcare sector, focusing on stocks such as Global Blood Therapeutics, Ionis Pharmaceuticals, and Akcea Therapeutics.

Crispr Therapeutics AG has an analyst consensus of Moderate Buy, with a price target consensus of $75.83, implying a 6.1% upside from current levels. In a report issued on November 25, Oppenheimer also maintained a Buy rating on the stock with a $80.00 price target.

See todays analyst top recommended stocks >>

The company has a one-year high of $74.00 and a one-year low of $22.22. Currently, Crispr Therapeutics AG has an average volume of 871.2K.

TipRanks has tracked 36,000 company insiders and found that a few of them are better than others when it comes to timing their transactions. See which 3 stocks are most likely to make moves following their insider activities.

CRISPR Therapeutics AG engages in the development and commercialization of therapies derived from genome-editing technology. Its proprietary platform CRISPR/Cas9-based therapeutics allows for precise and directed changes to genomic DNA. The company was founded by Rodger Novak, Emmanuelle Charpentier, and Shaun Patrick Foy in 2014 and is headquartered in Zug, Switzerland.

More here:
Crispr Therapeutics AG (CRSP) Receives a Buy from Wells Fargo - Analyst Ratings

In November 2018, He Jiankui, a researcher in Shenzhen, China, claimed he utilized CRISPR-Cas9 gene editing to alter the DNA of embryos for seven couples. A set of twins, whose names are Lula and Nana, were born. The research was met by global condemnation and calls for a moratorium on this type of gene editing.

However, aside from the public presentations and the resulting controversy and government crackdown on He Jiankui in China, the actual technical results have not been published. This week, the MIT Technology Review announced they had received a copy of Hes unpublished manuscript titled Birth of Twins After Genome Editing for HIV Resistance. They also received a second manuscript that discussed laboratory research on human and animal embryos. The research was apparently reviewed earlier by Nature and JAMA but is as yet unpublished. The MIT group had the papers reviewed by four experts, a legal expert, an IVF physician, an embryologist, and a gene-editing specialist.

MIT Technology Review writes, Their views were damning. Among them: key claims that He and his team made are not supported by the data; the babies parents may have been under pressure to agree to join the experiment; the supposed medical benefits are dubious at best; and the researchers moved forward with creating living human beings before they fully understood the effects of the edits they had made.

The analysis found 13 key problems, but one of the most relevant is that the stated purpose of the researchwhich is also under attackdid not occur. That is to say, He Jiankui and the authors of the research claim that the goal was to modify the CCR5 gene to make the children resistant to HIV. Not only did that not appear to have happened, but its questionable if it was the real reasonthe real, less scientific reason being to be the first scientists to modify human embryos using CRISPR gene that are then born.

One of the reviewers, Fyodor Urnov, a genome-editing specialist at the University of California Berkeley, said, The claim they have reproduced the prevalent CCR5 variant is a blatant misrepresentation of the actual data and can only be described by one term: a deliberate falsehood. The study shows that the research team instead failed to reproduce the prevalent CCR5 variant.

Another of the reviewers, Rita Vassena, scientific direct or the Eugin Group in Spain, noted, Approaching this document, I was hoping to see a reflective and mindful approach to gene editing in human embryos. Unfortunately, it reads more like an experiment in search of a purpose, an attempt to find a defensible reason to use CRISPR/Cas9 technology in human embryos at all costs, rather than a conscientious, carefully thought through, stepwise approach to editing the human genome for generations to come.

Vassena goes on to say, The idea that editing-derived embryos may one day be able to control the HIV epidemic, as the authors claim, is preposterous.

Hank Greely, professor of law at Stanford University, agreed with that assessment, calling it ludicrous. He also noted, We have no, or almost no, independent evidence for anything reported in this paper. Although I believe that the babies probably were DNA-edited and were born, theres very little evidence for that. Given the circumstances of this case, I am not willing to grant He Jiankui the usual presumption of honesty.

There is also significant doubt as to whether the parents involved in the study understood the procedures and the lack of necessity for having it performed. The father was HIV seropositive and it was under control using anti-retroviral therapy. There was little or no risk of HIV being transferred to the children under a normal in vitro fertilization treatment.

Jeanne OBrien, reproductive endocrinologist with Shady Grove Fertility in Atlanta, said, Being HIV-positive in China carries a significant social stigma. In spite of intense familial and societal obligations to have a child, HIV-positive patients have no access to treatment for infertility. The social context in which the clinical study was carried out is problematic and it targeted a vulnerable patient group. Did the study provide a genetic treatment for a social problem? Was this couple free from undue coercion?

Another problem, one of many, is that the gene edits performed were not the same as the mutations that are known to confer natural HIV resistance. The researchers indicated in the papers that they expect the edits to confer HIV resistance, because they are similar but not identical to CCR5 delta 32, the natural mutation that confers resistance. One of the babies only had edits to one of the CCR5 genes, which at best would only provide partial HIV resistance.

Greely said, Successfully is iffy here. None of the embryos got the 32-base-pair deletion to CCR5 that is known in millions of humans. Instead, the embryos/eventual babies got novel variations, whose effects are not clear. As well, what does partial resistance to HIV mean? How partial? And was that enough to justify transferring the embryo, with a CCR5 gene never before seen in humans, to a uterus for possible birth?

There is also significant concern, as there often is with CRISPR, of possible off-target edits. Urnov called it an egregious misrepresentation of the actual data that can, again, only be described as a blatant falsehood. It is technically impossible to determine whether an edited embryo did not show any off-target mutations without destroying that embryo by inspecting every one of its cells. This is a key problem for the entirety of the embryo-editing field, one that the authors sweep under the rug here.

In short, review of He Jiankuis research finds it deeply flawed in terms of rationale, procedures and results, with what can only be described as serious ethical problems and what appears to be a deliberate misrepresentation of the facts.

Originally posted here:
Reviewers of Chinese CRISPR Research: "Ludicrous" and "Dubious At Best" - BioSpace

DUBLIN--(BUSINESS WIRE)--The "Global CRISPR Technology Market 2019-2025" report has been added to ResearchAndMarkets.com's offering.

The global CRISPR technology market is expected to witness a significant growth rate during the forecast period. CRISPR technology enables the alteration of DNA sequences and modifies the gene function. CRISPR technology can be used for various applications such as treatment of diseases, correcting gene, crop modification, bio-fuel manufacturing and many more. Industry giants are working in CRISPR technology development and are investing in the R&D of the technology. Such investments are expected to create an opportunity for the growth of the market in the near future.

The global CRISPR technology market is segmented on the basis of application and end-user. Based on the application, the market is segmented into biomedical applications, agricultural applications, and industrial applications. In fruit crops, CRISPR technology has numerous applications as it improves the important agronomic traits such as biotic and abiotic stress tolerance and fruit quality. Further, on the basis of end-user, the market is segmented into pharmaceutical & biopharmaceutical companies, and academic & research institutes. CRISPR being a really new technology seeks the interest of everyone from doctors to academic & research institutes. CRISPR holds a lot of hidden potentials to cure many rare and incurable diseases that are still to be discovered and is driving the academic & research institutes as an end-user segment to grow with a significant rate in the market.

Geographically, the market is segmented into four major regions: North America, Europe, Asia-Pacific and Rest of the world (RoW). Among these, North America is expected to hold a prominent position in the global CRISPR technology market. The presence of major pharma companies in the region tends to enhance the growth of the global CRISPR market. Further, the report covers the analysis of several players operating in the market.

Some of the players include Thermo Fisher Scientific Inc., Merck KGaA, GenScript Biotech Corp., Horizon Discovery Group PLC, CRISPR Therapeutics AG, and others.

Key Topics Covered:

1. Report Summary

1.1. Research Methods and Tools

1.2. Market Breakdown

1.2.1. By Segments

1.2.2. By Geography

2. Market Overview and Insights

2.1. Scope of the Report

2.2. Analyst Insight & Current Market Trends

2.2.1. Key Findings

2.2.2. Recommendations

2.2.3. Conclusion

2.3. Rules & Regulations

3. Competitive Landscape

3.1. Company Share Analysis

3.2. Key Strategy Analysis

3.3. Key Company Analysis

3.3.1. Thermo Fisher Scientific, Inc.

3.3.1.1. Overview

3.3.1.2. Financial Analysis

3.3.1.3. SWOT Analysis

3.3.1.4. Recent Developments

3.3.2. Merck KGaA

3.3.2.1. Overview

3.3.2.2. Financial Analysis

3.3.2.3. SWOT Analysis

3.3.2.4. Recent Developments

3.3.3. GenScript Biotech Corp.

3.3.3.1. Overview

3.3.3.2. Financial Analysis

3.3.3.3. SWOT Analysis

3.3.3.4. Recent Developments

3.3.4. Horizon Discovery Group PLC

3.3.4.1. Overview

3.3.4.2. Financial Analysis

3.3.4.3. SWOT Analysis

3.3.4.4. Recent Developments

3.3.5. CRISPR Therapeutics AG

3.3.5.1. Overview

3.3.5.2. Financial Analysis

3.3.5.3. SWOT Analysis

3.3.5.4. Recent Developments

4. Market Determinants

4.1. Motivators

4.2. Restraints

4.3. Opportunities

5. Market Segmentation

5.1. CRISPR Technology Market by Application

5.1.1. Biomedical Applications

5.1.2. Agricultural Applications

5.1.3. Industrial Applications

5.2. Global CRISPR Technology Market by End-User

5.2.1. Pharmaceutical and Biopharmaceutical Companies

5.2.2. Academic & Research Institutes

6. Regional Analysis

6.1. North America

6.1.1. United States

6.1.2. Canada

6.2. Europe

6.2.1. UK

6.2.2. Germany

6.2.3. Italy

6.2.4. Spain

6.2.5. France

6.2.6. Rest of Europe

6.3. Asia-Pacific

6.3.1. China

6.3.2. India

6.3.3. Japan

6.3.4. Rest of Asia-Pacific

6.4. Rest of the World

7. Company Profiles

7.1. AstraZeneca PLC

7.2. BASF SE

7.3. Beam Therapeutics Inc.

7.4. Bio-Rad Laboratories, Inc.

7.5. Caribou Bioscience Inc.

7.6. Cellectics SA

7.7. Cibus, Ltd.

7.8. CRISPR Therapeutics AG

7.9. Danaher Corp.

7.10. Editas Medicine

7.11. GeneCopoeia inc.

7.12. GenScript Biotech Corp.

7.13. Horizon Discovery Group PLC

7.14. Intellia Therapeutics Inc.

7.15. Lonza Group Ltd.

7.16. Merck KGaA

7.17. New England Biolabs, Inc.

7.18. Origene Technologies, Inc.

7.19. Pairwise Plants

7.20. Precision Bioscience, Inc.

7.21. Sangamo Therapeutics Inc.

7.22. Thermo Fisher Scientific, Inc.

7.23. Transposagen Biopharmaceuticals, Inc.

See the original post here:
Global CRISPR Technology Market Report 2019-2025 with Analysis of Key Companies - Thermo Fisher Scientific, Merck KGaA, GenScript Biotech, Horizon...

Chinese scientist He Jiankui shocked the world by claiming he had helped make the first gene-edited babies. One year later, mystery surrounds his fate as well as theirs.

He has not been seen publicly since January, his work has not been published and nothing is known about the health of the babies.

Thats the story its all cloaked in secrecy, which is not productive for the advance of understanding, said Stanford bioethicist Dr. William Hurlbut.

..

The report said the twins and people involved in a second pregnancy using a gene-edited embryo would be monitored by government health departments. Nothing has been revealed about the third baby, which should have been born from that second pregnancy in late summer.

Chinese officials have seized the remaining edited embryos and Hes lab records.

A moratorium is no longer strong enough, and regulation is needed, CRISPR pioneer Jennifer Doudna of the University of California, Berkeley recently wrote in acommentaryin the journal Science.

She noted that the World Health Organization has asked regulators in all countries not to allow such experiments, and that a Russian scientist recently proposed one.

Read full, original post: 1 year later, mystery surrounds Chinas gene-edited babies

See more here:
There's still a lot we don't know about China's controversial CRISPR babies, including their health status - Genetic Literacy Project

When Ben Campbell started out with The New York Produce Show and Conference, he was a professor at the University of Connecticut. During the event, Ben and his students participated in ourUniversity Interchangeprogram, which provides a forum for university professors to share cutting-edge research with the trade and thus help fulfill their mission to disseminate knowledge, while also providing an educational and mentorship program for students. He found the program so valuable, that when he moved on to the University of Georgia he asked if we would expand the program. Which we did.

Professor Campbell has presented on a number of important topics, including these:

Setting Producers Free Production Agriculture And The Regulatory Burden:Can States Help Northeast Production Thrive?Are They Inclined To Do So?

Perceptions And Misperceptions: Consumer Attitudes On Organic And Local University Of Connecticut Study To Be Unveiled At New York Produce Show and Conference

Connecticut Professor Ben Campbell Comes Back To The New York Produce Show With Seminal Work On Consumer Reaction To The Marketing Of Locally Grown Produce

We asked Pundit Investigator and Special Projects editor Mira Slott to see what the good professor has in store for attendees this year:

Ben CampbellAssistant Professor,Department of Agricultural andApplied Economics,University of Georgia

New York Produce Show attendees await your thought-provoking consumer research, measuring how different variables influence and impact produce shoppers perceptions and purchasing decisions.

In 2018, you delved into the multitude of ways consumers can access information to understand how various generations are getting information about produce, while also analyzing how these methods are impacting purchasing. You also have presented on organic and local. What do you have planned for this year?

A: This year, my research examines how giving consumers information about GMOs,CRISPR, organic and pesticides impacts their likelihood of purchasing different products.

Q: Thats a hot topic with the new federal and state labeling laws, increased pesticide restrictions, and the influence on retail buying

A: The emphasis is very much the new labeling laws coming into place with GMOs, and new technologies, and a lot of retail policies banning particular production methods. For instance, you seeneonicotinoidsbeing banned in Maryland, and these pollinators being phased out in Home Depot and Lowes of a lot or all of their plant stock. There are new pesticide bans in homes in Ontario, and within school grounds in Connecticut and New York. When looking at produce, there are issues with GMOs and pesticides in the production process as well.

We see bans of products in different areas, and a lot of misinformation about products. People often dont understand GMOs, but they are very much not in favor of them. So, youre against something but you dont know what it is. You see this phenomenon across the board in relation to different chemicals and pesticides too. And even with organic, misinformation abounds. Theres strong lobbying against something or for it and mixed messaging.

Q: How do you set out to understand the impacts of that cacophonous messaging?

A: I was interested in discovering if we gave people fact-based information about what these production methods are, how would that effect their perceptions or influence their potential purchase of these foods, plants and turf, which use these production practices.

Can information impact the role of production practices on the decision to purchase, and more specifically the type of message and source of message.

One of our objectives was the premise that consumer sentiment drives policy changes at the federal, state and local levels, and at retail, and how these decisions could translate into policy implications.

Q: Could you describe the study methodology. What types of production practices were included and what products? What information did you provide participants?

A: We gave participants different treatments and defined the technology of each, looking at three segments: food, plants and turf. The production practices included insecticides, fungicides, herbicides, CRISPR, GMO and organic. Heres the meaning of what this is and the information on how this production practice works.

Q: Were these scientific definitions? How detailed or complex were the descriptions? Were they consumer-friendly?

A: We purposefully adhered to scientific information for the different treatments, though at a level that non-scientists can understand.

Editors note: here are the definitions provided to participants:

*CRISPR:a new biotechnology that allows scientists to directly edit an organisms genetic material (DNA). This does not require transferring DNA from one organism to another.

*Genetically-Modified Organism (GMO):an organism in which the genetic material (DNA) has been altered through the use of modern biotechnologies. The alteration typically involves transferring DNA from one organism to another.

*Insecticide:a pesticide that is used to eliminate or repel insects.

*Fungicide:a pesticide that is used to eliminate or prevent the growth of fungi, molds, and their spores.

*Herbicide:a pesticide that is used to eliminate or prevent the growth of plants

*Organic:the application of a set of cultural, biological, and mechanical practices that support the cycling of on-farm-resources, promote ecological balance, and conserve biodiversity. These include maintaining or enhancing soil and water quality, conserving wetlands, woodlands, and wildlife and forbidding use of synthetic fertilizers, sewage sludge, irradiation and genetic engineering.

Q: These scientific definitions arent really that appealing when talking about food! Did people understand these terms, or want further explanation or context on usage? The GMO and CRISPR definitions dont seem likely to allay a consumers pre-disposed concern about the effect these technologies could have on a food item. Although, I could see the draw of wanting to purchase produce void of insects, fungi and mold

A: Some people got it. Some people didnt. Most people have never heard of CRISPR. We divvied people into groups to determine how likely they would be to buy the product based on the information we provided them on the production practices of that product.

Q: How were the groups formed?

A: Respondents were randomly divided into the treatment groups. After being randomly placed into a treatment group, they were given the information treatment associated with that treatment group.

We had a control group that didnt receive any information, we just said CRISPR or GMO

Q: Who participated in the study, the numbers, demographics, how did you glean peoples preferences, etc.

A: We did an online survey of 2,500 respondents in the spring/summer of 2019. It included Southeastern US residents in nine states with 10-20 percent of participants per state. The average age was 44 and the average income was $50,000. We had 64 percent female and 71 percent Caucasian.

Q: Did you assess the participants knowledge, or perceived knowledge, of each practice going into the study for a relative comparison? Did you break down the sample further to determine, for example, if some consumers were more prone to buy organic

A: Not exactly in that way, but we asked about knowledge level, how knowledgeable are you about organicand I will show the effects of that in our model.

Q: So, this is the knowledge level that is perceived by the participant, not necessarily the correct knowledge?

A: Yes, thats an important point. We know the odds are that consumers are not correct in their knowledge of the production practices, but perception is the reality. We wanted to find out their perceived knowledge of the different production practices. With 0 = No Knowledge; 50 = Somewhat Knowledgeable; and 100 = Extremely Knowledgeable.

Organic was the highest rated. People said they had the most knowledge of it at 64 for food, 62 for plants, and 60 for turf. Organic stood out relative to all the other practices, where there was much less fluctuation. This compared to both GMO and CRISPR at 44 for food, 44 for plants, and 45 for turf. Insecticides 42 for food, 47 for plants and 48 for turf; herbicide 45 for food, 47 for plants, and 48 for turf; and fungicide, 43 for food, 45 for plants and 48 for turf.

Q: What can be learned from this?

A: Interestingly, people indicated they were more knowledgeable about pesticides as they are applied to turf but less knowledgeable as they are applied to their food, though we see moves to eliminate pesticides on food even though people say they are not that knowledgeable. From a produce standpoint, this is informative, first because consumers are not knowledgeable, and second because consumers who are not knowledgeable are making demands on produce production methods or are not able to knowledgably engage with activists in the discussion.

Q: Since youll be presenting this information to executives in the fresh produce industry, what was included in the food category? Did you distinguish between fresh produce and processed items?

A: In looking at the food category as a whole, by and large its going to be an ingredient, a GMO or CRISPR is going to be applied at the production level, so its not directly targeting produce.

Q: The reason I ask,Brad Rickard of Cornell, a veteran like you at our New York Produce Shows, has presented fascinating research related to this topic.In one studyhe conducted, consumer acceptance of GMOs changed based on the food category (grain crop, fruit or beef) and the level of processing. For instance, consumers showed more willingness to buy the GM processed products relative to their GM fresh versions.

A: In our study, were focused on the impact of messaging. So, in this case, if youre more accepting of a treatment in processed food, would knowledge of the treatment push you to be more accepting? Similarly, if youre less accepting of a treatment in fresh produce, will knowledge make you more accepting. Conversely, will more information about insecticides, herbicides, and fungicide usage on conventional produce increase acceptance, and could that lessen acceptance of organic, which restricts that use?

Q: So, the scale of acceptance might change on processedversusfresh, but thats not what youre measuring in this study

A: Were interested to see if our messaging is going to nudge a consumers acceptance and willingness to purchase one way or the other; thats what were focused on in this study.

Q: Thats interesting, because now youre looking at a domino effect of how knowledge of one type of production practice could change acceptance of products in several categories

A: What we found, in fact, is if we showed participants information about pesticides the message that pesticides are used to eliminate insects or fungicides and the growth of fungi, molds, and their spores or herbicides to get rid of the problem plant if you got that message, you rated organic lower.

If its an apple or a processed apple, I dont think the differences will be that big. The result is still moving in the same direction. Receiving the information on pesticides nudges the participants on likeliness to purchase conventional over organic.

Q: How are you defining nudge? Could there be a noticeable change in purchasing choices if consumers received such messaging?

A: Messaging can have marginal impacts. The lesson here is around the messaging and consumer knowledge actually, perceived knowledge. If I think I know more about CRISPR, Im more accepting of buying that product. So, this is about getting the information out there. That information impacts how we view these things.

If someone views pesticides as bad, providing an explanation negates some of the mysteries of pesticides I understand why theyre using it, and Im less likely to buy organic and more likely to buy something produced with insecticides. We see this movement here. This messaging can impact the perceived value of different products, and the effects on each other.

Q: Doesnt it get complicated, though, since there are so many different types of pesticides, and regulations, safe levels of use, etc., and sometimes frightening media reports can lump them all together?

A: Organic is often viewed as less risky for that reason.

Q: If someone is a diehard organic shopper, they might not be as flexible to change

A: Youre not changing people on the endpoints, youre changing people in the middle. Were looking at the average person, not those on the extremes. Youre not changing the person who is diehard organic, or diehard anti-organic. Their minds are set. Youre shifting perceptions of those in the middle.

Q: How important is pricing in this equation? Did you consider this? For example, isnt there a correlation of organic produce purchases increasing when the pricing is more on par with the conventional counterpart?

A: Yes, pricing certainly could have an impact. For this study, we wanted everything else to be constant, to control for what the message was when consumers were considering acceptance of the different treatments. If an herbicide is used or an insecticide is used, what is the effect of that on consumer purchase decisions?

We chose to give participants a scientific definition instead of doing something more general. The reason we did this is, in the marketplace you see a lot of different labels, and best practice claims, both good and bad. We wanted to see if we gave consumers fact-based information, might that affect their views on these foods that have these different characteristics?

Q: Did you consider homing in on particular products, such as an Arctic apple, where the genetically engineered practice prevents browning, and how that quality might change a consumers likelihood of purchasing it? Also, what if you used a more consumer-friendly description of the production process with clever marketing as can be found on theArctic apple website?

A: These variables certainly could play a role We intentionally kept the definitions scientific to alleviate the positive and negative biases.

Q: You note the broad scope of labeling, certifications and product claims vying for consumer attention. Youve done other studies analyzing consumer acceptance and perceptions or misperceptions of these various labels (environmental labeling, local, organic, natural, bee-friendly, etc.). For instance, I remember in one study, you found that some consumers thought that local and organic were interchangeable. Are you taking these types of influences into account when youre conducting your analysis?

A: We touch on them, and will discuss them in our presentation, but by and large, were going to stick with the production processes and focus on the ones coming under threat, like GMO, and CRISPR. Local is not one of those, because its here to stay. With respect to environmentally friendly labels, those are out there, but we can see that insecticides, fungicides, herbicides, GMO, and CRISPR are the ones really on the forefront now of consumer protests and where people have strong opinions.

Q: Based on your study parameters and methodology, what did you learn? What is most illuminating for produce executives?

A: I can give you some examples from the charts Ill be presenting

With no information, the likeliness to purchase organic was 60, but when given information about pesticides, your score for organic went down to 57. Thats a 3 percent drop in your likeliness to purchase a product, so thats what were seeing.

We see that giving consumers information about pesticides had a small effect on their likeliness to purchase organic. When you hear messaging now, you hear that organic is better because no pesticides are used. But we know that consumers indicate they are not knowledgeable about pesticides. When we give them minimal information about pesticides, they move slightly away from organics.

That example was for turf. For food, it went from 64 to 62.

Q: Is that statistically relevant?

A: A two percent shift doesnt sound like a lot, but were seeing these differences here, which could move people in the middle. Though it sounds marginal, we know that for many consumers, production method makes up a small part of their purchasing decision. If you weaken the case for organic, then it will decrease the likelihood of paying a higher price for organic and thereby lower the probability of purchasing organic.

Q: What movement occurs when you introduce CRISPR and GMO information?

A: For CRISPR and GMO, showing CRISPR info moved the rating from 44 (no info) to 46 (CRISPR info); for GMO: 44 (no info) to 42 (CRSPR info); GMO: 44 (no info) to 41 (GMO info). This indicates that providing GMO info hurts GMO purchasing; GMO: 44 ( no info) to 46 (all info) this implies when looking at all the different methods, GMO is not seen as bad; CRISPR: 44 (no info) to 46 (CRISPR info) -providing CRISPR info helps with purchasing; CRISPR: 44 (no info) to 40 (GMO info) GMO info causes a negative reaction to CRISPR.

Q: If you add in other variables of shopping in a grocery store, the changes could be much more significant

A: Thats right. Two years ago, we examined price premiums and how pricing could impact the market. Once you start adding or subtracting premiums or discounts, that makes a difference. Even if youre at a higher price, I can generate or negate that price premium, through messaging impacts.

We had five percent of the group that would not purchase food with insecticides. These were diehards on the endpoints. And when we gave the group the information on insecticides, that 5 percent of diehards dropped to 2 percent that would not purchase the product. We are talking a 3 percent swing, or getting them from, Im not buying, to being open to buying now.

Q: Did you break down the results by demographics?

A: There are things going on demographic-wise. We see with GMOs, older people are less likely to buy GMOs. People in rural areas didnt like GMOs as much, but by and large, the big thing is, the more people perceive they know about these production practices, the more likely they are to consider buying these products with these practices.

Q: But you cant really control peoples perceptions, or whether they grasped the scientific definition you provided The type of information and the way its communicated seems to be very important.

A: Yes. It depends on what you want to do. If youre selling organic, you dont want to give people the scientific information on pesticides. If youre selling something traditional, non-organic, providing information about production practices and why youre doing it can be a way to counter organics.

Its the same with CRISPR and GMO, in terms of how consumers perceive your product, and in turn other products in a different way.

Q: This goes back to all the information out there that is not always scientific and scares people Some of that information can be quite impressionable. One of the issues magnified by vocal GMO opponents is the unknowns, or the long-term potential negative effects of consumption of GMOs Your definition of CRISPR and GMO outlines scientifically how the practice works, but doesnt address the impacts these alterations could have, either positive or negative. For instance, there is no mention of the positive attributes of the Arctic apple non-browning trait.

A: The key is the information you give will be a nudge to the average consumer, more so than those on the endpoints. The information you give can be powerful. It can have a meaningful effect on those people in the middle who may have strong opinions but can be influenced.

Q: To clarify, you just asked consumers about food, in general, without segmenting food categories, like fruits and vegetables, and you didnt give them examples?

A: No, we didnt. The problem is there are so many examples and categories. If the notion is your likeliness to buy a GMO apple is less than a GMO Pop Tart, the nudge to change that likeliness is what were interested in measuring based on receiving information on the production practice of GMO. Youll see that nudge going in the same direction with either one. I can spin it toward produce or food, in general, but the message stays the same; its just the starting point differs, as Brad Rickard showed in his study that you referenced earlier. Could we ask about produce directly, yes, but then you lose the process we set for this study.

Q: Miguel Gmez of Cornell will be revealing his latest peer-reviewed research, which will be hot off the press, at the NYPS, related to potential impacts of new GMO labeling requirements and non-GMO labeling counter-plays. It specifically targets strawberries, potatoes and apples. [Editors note, a sneak preview Q&A piece is here].

A: Im looking forward to seeing his presentation. I have a study, now in review, looking at tomatoes and tomato plants with GMO and non-GMO labels, and no labels, and those differences, and as a plant or as the product you buy at the supermarket to eat. The differences of how consumers perceive these things is there.

Labeling and how you message matters how you message can impact how consumers feel about your products and others products. So, if youre giving people good information about pesticide usage, it could have a positive impact on peoples views of your product if youre using those practices, and a detrimental impact on organic purchases. If you provide information de-mystifying GMO or CRISPR, it could have a negative effect on purchases of non-GMO, or non-CRISPR products. Thats the point Im trying to get across.

This messaging is interdependent. If you put a GMO label on one thing, it could have a detrimental impact on something else.

Q: Are you interested in extending this study in anyway? For instance, taking it from surveys to an actual supermarket setting, or as youve done in other instances, arrange scenarios where youre interviewing consumers in person and presenting them with actual products

A: Thats always the ideal, but its challenging to conduct studies like that. The problem is getting access to stores to do it. Retailers sometimes dont like you telling consumers about the products in the stores and giving them different messages about pesticides, GMOs, and CRISPR. That can be tricky, so a lot of times we stick to the online surveys or conducting our research outside of stores. I would love to go into stores and change prices of organic products and see how demand changes

Where we go from here the next step Im working on a study looking at plants and the media message, whats the source of the information, and how that impacts consumer perceptions. I would love to understand if the information is from a producer versus a retailer versus an association, versus a mass media outlet. What would happen if each of these players told you the same message, how would that influence the different choices? If an ag group told you about GMOs versus a retailer telling you about GMOs, would that impact the message and your purchasing decision?

Q: That research sounds fascinating

A: My hypothesis would be if the information was coming from an activist group, the consumer would take the information more seriously generally people trust universities; they dont trust the government that much. They trust activists, they dont trust retailers as much; they dont trust industry associations as much. You have these different groups that are vying for power and influence, in respect to getting information out.

Q: Its an interesting hypothesis to test. Will consumers be more accepting of a scientific-based, academic study debunking theEWG Dirty Dozenclaims than if they are warned to stay away from produce on the Dirty Dozen list on the Today Show or Dr. Oz? Further, if the messenger of that scientific-based study is theAlliance for Food and Farming, an industry organization, will that influence the consumers perception of its accuracy?Weve been reporting on these issues for many years.

A: At the NYPS, Ill talk about the production-practices messaging study we did, and the next steps

If you put a government GMO label on a product, it may have a detrimental impact on the consumers desire to purchase that product. But what we found in this study was if the GMO label also comes with good information describing what it is, the consumers could be nudged toward that GM product, and affect how they view other products. In the same way, pesticides are not all bad when you tell consumers what they are, and why theyre used. They may move incrementally toward the pesticide group, especially with plants, and away from the organic alternative. When people understand what it is, they are less likely to fear it and thereby say they are more likely to buy it.

Q: Did any of the results surprise you?

A: I expected if you provided pesticide information, it was only going to have an impact on pesticide products. I wasnt expecting to see so much cross effect on the other products, in respect to pesticides impacting organic, and GMOs impacting CRISPR and organic.

Read more here:
The Science of Product Messaging: How Unbiased Information on Pesticides, Fungicides, Herbicides, Organics, GMO and CRISPR Can Affect Consumer Buying...

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Global and Regional CRISPR And CRISPR-Associated (Cas) Genes Market 2019 by Manufacturers, Regions, Type and Application, Forecast to 2025 - Breakaway...

What happened

Shares of CRISPR Therapeutics (NASDAQ:CRSP) gained over 42% last month, according to data provided by S&P Global Market Intelligence. The company provided the first glimpse of CTX001, a therapy based on CRISPR gene editing, in sickle cell disease and transfusion-dependent beta-thalassemia (TDT). While the preliminary results were from only two patients -- one in each indication -- they demonstrated promising potential for the approach.

Investors cheered the update, which was the first from any company developing CRISPR-based tools. The news sent several gene-editing stocks higher. Shares of Editas Medicine and Intellia Therapeutics rose as well, although super-early success for CTX001 means next to nothing for industry peers. More meaningful might be the implications for Vertex Pharmaceuticals (NASDAQ:VRTX), the collaboration partner of CRISPR Therapeutics for CTX001, which could be handsomely rewarded for its early bet on the gene-editing technique.

Image source: Getty Images.

CRISPR Therapeutics reported that after one dose of CTX001, the individual with TDT was transfusion independent through the nine-month mark. The patient had averaged 16.5 blood transfusions per year in the two years prior to the clinical trial.

Similarly, after one dose of CTX001, the individual with sickle cell disease was free of vaso-occlusive crises (painful blockages of small blood vessels caused by abnormally shaped blood cells) at the four-month mark. The patient had averaged seven such events in the two years prior to the study. Both patients achieved promising results for an important biomarker as well.

How excited are investors? Well, CRISPR Therapeutics announced a public stock offering days after providing the clinical update, but after briefly tumbling, shares actually shook off the dilution concerns and continued ascending through the end of the month. The business ended September with over $629 million in cash and raised up to $315 million from the November offering.

Simply put, the early update from CTX001 is about as good as investors could have hoped for, although the results are obviously very preliminary. The studies are designed to enroll dozens of patients and track them for two years. Will the results prove durable? Will adverse events or other safety issues crop up as the studies mature? Investors will have to remain patient, but CRISPR Therapeutics remains the top gene-editing stock.

Read more:
Here's Why CRISPR Therapeutics Jumped 42.2% in November - The Motley Fool

Earlier this year a source sent us a copy of an unpublished manuscript describing the creation of the first gene-edited babies, born last year in China. Today, we are making excerpts of that manuscript public for the first time.

Titled Birth of Twins After Genome Editing for HIV Resistance, and 4,699 words long, the still unpublished paper was authored by He Jiankui, the Chinese biophysicist who created the edited twin girls. A second manuscript we also received discusses laboratory research on human and animal embryos.

The metadata in the files we were sent indicate that the two draft papers were edited by He in late November 2018 and appear to be what he initially submitted for publication. Other versions, including a combined manuscript, may also exist. After consideration by at least two prestigious journals, Nature and JAMA, his research remains unpublished.

The text of the twins paper is replete with expansive claims of a medical breakthrough that can control the HIV epidemic. It claims successa word used more than oncein using a novel therapy to render the girls resistant to HIV. Yet surprisingly, it makes little attempt to prove that the twins really are resistant to the virus. And the text largely ignores data elsewhere in the paper suggesting that the editing went wrong.

We shared the unpublished manuscripts with four expertsa legal scholar, an IVF doctor, an embryologist, and a gene-editing specialistand asked them for their reactions. Their views were damning. Among them: key claims that He and his team made are not supported by the data; the babies parents may have been under pressure to agree to join the experiment; the supposed medical benefits are dubious at best; and the researchers moved forward with creating living human beings before they fully understood the effects of the edits they had made.

Because these documents relate to one of the most important public interest issues of all timethe ability to change human heredity using technologywe here present excerpts from the twins manuscript, together with some of the experts comments, and explain the questions they raise. The excerpts are in the order in which they appear in the paper.

To understand why the manuscripts have remained unpublished up to now, read the accompanying article on He's attempts to get them into scientific journals. For the case for making their content public, read the op-ed by Kiran Musunuru, a gene-editing specialist at the University of Pennsylvania, who argues the Chinese data shows that gene-editing for reproduction is unsafe and premature.

1. Why arent the doctors among the papers authors?

The manuscript begins with a list of the authors10 of them, mostly from He Jiankuis lab at the Southern University of Science and Technology, but also including Hua Bai, director of an AIDS support network, who helped recruit couples, and Michael Deem, an American biophysicist whose role is under review by Rice University.

Its a small number of people for such a significant project, and one reason is that some names are missingnotably, the fertility doctors who treated the patients and the obstetrician who delivered the babies. Concealing them may be an attempt to obscure the identities of the patients. However, it also leaves unclear whether or not these doctors understood they were helping to create the first gene-edited babies.

To some, the question of whether the manuscript is trustworthy arises immediately.

Hank Greely, professor of law, Stanford University: We have no, or almost no, independent evidence for anything reported in this paper. Although I believe that the babies probably were DNA-edited and were born, theres very little evidence for that. Given the circumstances of this case, I am not willing to grant He Jiankui the usual presumption of honesty.

2. The researchers own data dont support their main claims

The abstract, or summary, lays out the aim of the projectto generate humans resistant to HIVand the main results. It states that the team was successfully able to reproduce a known mutation in a gene called CCR5. The small percentage of people born naturally with this mutation, known as CCR5 delta 32, can be immune to infection by HIV.

But the summary goes well beyond what the data in the paper can back up. Specifically, as well see later, the team didnt actually reproduce the known mutation. Rather, they created new mutations, which might lead to HIV resistance but might not. They never checked to see, according to the paper.

Fyodor Urnov, genome-editing scientist, Innovative Genomics Institute, University of California, Berkeley: The claim they have reproduced the prevalent CCR5 variant is a blatant misrepresentation of the actual data and can only be described by one term: a deliberate falsehood. The study shows that the research team instead failed to reproduce the prevalent CCR5 variant. The statement that embryo editing will help millions is equal parts delusional and outrageous, and is akin to saying that the 1969 moonwalk brings hopes to millions of human beings seeking to live on the moon.

Rita Vassena, scientific director, Eugin Group: Approaching this document, I was hoping to see a reflective and mindful approach to gene editing in human embryos. Unfortunately, it reads more like an experiment in search of a purpose, an attempt to find a defensible reason to use CRISPR/Cas9 technology in human embryos at all costs, rather than a conscientious, carefully thought through, stepwise approach to editing the human genome for generations to come. As the current scientific consensus indicates, the use of CRISPR/Cas9 in human embryos destined to give rise to a pregnancy is, at this stage, unjustified and unnecessary, and should not be pursued.

3. Gene-editing embryos wont bring HIV under control, especially in the worst-affected countries

The end of the abstract and beginning of the main text is where the authors justify their research. They suggest that gene-editing babies could save millions of people from HIV infection. Our commenters call this claim preposterous and ludicrous, and point out that even if the CRISPR method works to create people who are HIV resistant, its unlikely to be practical in places where HIV is rampant, such as in the southern part of Africa.

Rita Vassena: This work offers little justification for the editing and subsequent transfer of human embryos to generate a pregnancy. The idea that editing-derived embryos may one day be able to control the HIV epidemic, as the authors claim, is preposterous. Public health initiatives, education, and widespread access to antiviral drugs have been shown to control the HIV epidemic.

Hank Greely: That this is a plausible way to control the HIV epidemic seems ludicrous. If every baby in the world were given this variation (beyond unlikely), it would begin to affect HIV infection substantially in 20 to 30 years, by which time we should have much better methods of stemming the epidemicas well as existing methods that have substantially, if not yet sufficiently, slowed it. The 64% increase in infections in China (if true) is from a very low base. China has a substantially lower rate of HIV infection than Western countries. The situation in some developing countries remains more serious. But that this high-tech response is likely to be helpful in those countries is not plausible.

4. The parents might have wanted to take part for the wrong reasons

Contrary to some interpretations, the point of using CRISPR on the babies DNA wasnt to prevent them from catching HIV from their father, who was infected. As the paper describes, this was achieved by sperm washing, a well-established technique. Instead, the purpose of the editing was to give the children immunity to HIV later in life. Thus, the experiment didnt provide clear, immediate medical benefits to either the parents or the children. Why did the couple agree? One reason may have been to access fertility treatment at all.

Rita Vassena: I find it worrying that the husband in the couple offered this experimental genome editing was positive to HIV infection, as one can imagine the unnecessary emotional pressure on the couple to consent to a procedure offering no improvement to the patient and their childrens health, but carrying a potential risk of negative consequences. It is worth remembering that HIV infection is not passed on through generations like a genetic disease; the embryo needs to catch the infection. For this reason, preventive measures such as controlling the viral load of the patient with appropriate drugs, and careful handling of the gametes during IVF, can avoid contagion very efficiently. Current assisted reproductive techniques ensure safe procreation for HIV-positive men and women, avoiding both horizontal (between partners) and vertical (between parent and embryo/fetus) transmission, making the editing of embryos in these cases unnecessary. In fact, the couple in the experiment did undergo such ART procedures, consisting in this case of an extended wash of semen to remove all seminal fluid, which may harbor HIV. Extended sperm washing has been used for almost two decades in IVF laboratories worldwide and in thousands of patients; in ours and others experience, it is safe for both parents and their future children and does not entail invasive manipulation of embryos.

Jeanne OBrien, reproductive endocrinologist, Shady Grove Fertility: Being HIV-positive in China carries a significant social stigma. In spite of intense familial and societal obligations to have a child, HIV-positive patients have no access to treatment for infertility. The social context in which the clinical study was carried out is problematic and it targeted a vulnerable patient group. Did the study provide a genetic treatment for a social problem? Was this couple free from undue coercion?

5. The gene edits werent the same as the mutations that confer natural HIV resistance

Here, the researchers describe the changes CRISPR actually made to the twins. They removed a few cells from the IVF embryos to look at their DNA, and found that edits intended to disable the CCR5 gene had indeed taken hold.

But while they expect these edits to confer HIV resistance by nullifying the activity of the gene, they cant know for sure, because the edits are similar but not identical to CCR5 delta 32, the mutation that occurs in nature. Moreover, only one of the embryos had edits to both copies of the CCR5 gene (one from each parent); the other had only one edited, giving partial HIV resistance at best.

Hank Greely: Successfully is iffy here. None of the embryos got the 32-base-pair deletion to CCR5 that is known in millions of humans. Instead, the embryos/eventual babies got novel variations, whose effects are not clear. As well, what does partial resistance to HIV mean? How partial? And was that enough to justify transferring the embryo, with a CCR5 gene never before seen in humans, to a uterus for possible birth?

6. There could have been other, unwanted CRISPR edits

CRISPR isnt a perfect tool. Trying to edit one gene can sometimes create other, unintended changes elsewhere in the genome. Here the team discusses their search for such unwanted edits, called off-target mutations, and say they found just one.

The search was incomplete, however, and the manuscript also glosses over a key point: any cells the researchers took from the early-stage embryos to test didnt, therefore, actually contribute to the twins bodies. The remaining cells, the ones that would multiply and grow to become the twins, could have harbored off-target effects too, but there would have been no way to know that in advance of starting the pregnancy.

Fyodor Urnov: An egregious misrepresentation of the actual data that can, again, only be described as a blatant falsehood. It is technically impossible to determine whether an edited embryo did not show any off-target mutations without destroying that embryo by inspecting every one of its cells. This is a key problem for the entirety of the embryo-editing field, one that the authors sweep under the rug here.

7. The doctors treating the couple may not have known what was going on

Reporting by a variety of news outlets, including the Wall Street Journal, has charged that Hes team tricked doctors by switching blood samples and that not all of them knew they were involved in creating gene-edited children. If true, thats a problem, since its the duty of doctors to do what is in the best interest of the patient.

Jeanne OBrien: The IVF procedure described follows the same steps and time line whether or not CRISPR is used for genome editing. The Chinese physicians who performed the IVF may have been unaware of the fathers HIV status or that the embryos were genetically modified. He Jiankui would have only needed a willing embryologist to inject CRISPR at the time of insemination. Hes comments make it appear as if the physicians who performed the IVF were not involved in the subsequent decision regarding which embryos to select for transfer. This is a wake-up call to physicians involved in IVF: the science and technology will continue to progress, and desperate couples with infertility may overlook the unknowns or believe the technology is proven safe. Once we, the infertility physicians, knowingly transfer an embryo with germline editing, we are in essence confirming the safety of the modification to the parents and the future child. Is it ever possible to know that?

8. The manuscript misrepresents when the babies were born

By now, several media reports and people familiar with the research have established that the twins were born in October, not November. Why did Hes team include a false date? It may have been to protect the anonymity of the patients and their twins. In a country the size of China, there could be more than ten thousand sets of twins born each month. The falsified date may have been an attempt to make their reidentification even more difficult.

9. Its not clear if there was a proper ethics review

The paper includes an exceptionally brief discussion of ethics. It says the research plan was registered with the China Clinical Trial Registry, but in fact the public registration occurred only after the twins were born.

Hank Greely: Registered when? The answer is on November 8, 2018, after the births and very shortly before they were announced, and probably in order to increase publication potential. This was not a normal registration. Maybe there was an ethics approvalthough that hospital has denied it. Who is telling the truth? Not sure well ever know. The phrase we were told about a comprehensive ethics review is not very powerful evidence. The article also does not discuss the Chinese ban on assisted reproductive services for HIV-positive parents. It has been reported that He had other men pretend to be the intended fathers for purpose of the required HIV tests. The article doesnt say this. It seems to me likely to be trueand damning. If true, it means He defrauded the Chinese regulatory process.

10. The researchers didnt test whether the HIV immunity worked before creating living human beings

Here the Chinese team outlines their plan to collect blood from the twins to see if their edited cells really resist HIV. That is something they could have tried to learn ahead of time, before creating the girls. Before transferring the embryos, they could have kept them frozen while they made identical edits in laboratory cells and tested the effects of HIV on those cells.

Fyodor Urnov: This statement proves that the research team placed their interests above those of the couple who donated the embryos and of their prospective children. There is zero evidence in the manuscript supporting the essential expectation that the new forms of CCR5 would be HIV-protective. It was essential to have determined that before the embryos were implanted. They could have done so using a known assay: introduce the same edits into immune system cells in the laboratory and then infect them with HIV. Only the cells that have HIV-protective variants of CCR5 survive. The research team chose not to do that assay. Instead, they made children out of embryos that had forms of CCR5 of entirely uncertain functional impact. Were the researchers in a rush? Did they simply not care? Whatever the explanation, this egregious violation of elementary norms of ethics and of research borders on the criminal.

11. An American Nobelist may have helped He justify his experiment

The articles conclusion contains an unexpected digression that puts forth an entirely new justification for the research, one that connects the project to the heart of the HIV epidemic in Africa. Its that many uninfected children of African mothers with HIV suffer a syndrome called HEU that makes them more susceptible to a variety of childhood illnesses. The authors say genome editing could be a novel strategy against HEU.

There isnt any evidence for this idea, but there are some clues about where He got it. In an email he sent on November 22 to Craig Mello, a biologist at the University of Massachusetts who at the time was an advisor to one of his companies, He thanked Mello for suggestions on the topic and enclosed in his email the same paragraph above.

Does that mean Mello, a winner of the 2006 Nobel Prize for medicine, contributed a key idea to the paper? Mello was told about the twins project early on but, through a spokesman, says he never gave He advice on how to write the paper. According to Hes email, however, any such interaction was meant to remain unacknowledged. Again, I wont tell people you know what is happening here, he wrote to Mello.

12. The project had other supporters, but some key information is missing

The manuscript concludes by thanking a list of people who, according to He, gave him direct feedback on draft versions of the text or other advice. In an acknowledgement for editing the text, he names Mark Dewitt, a researcher at the University of California. Dewitt didnt reply to emails but earlier gave a description of his role, saying he had warned against the project. William Hurlbut, an ethicist at Stanford, says he gave ethics advice to He but didnt know that the Chinese scientist had created children.

He also thanks W.R. Twink Allen, an equine reproduction specialist in the United Kingdom, and Allens onetime student Jin Zhang, also known as John Zhang, who is now head of New Hope Fertility Center in New York, one of the largest in the US. According to reports, Zhang was planning with He late last year to open a medical tourism business for gene-edited babies.

Of these names, only Allens has not previously been cited in connection with the CRISPR-baby research. Allen did not reply to attempts to contact him by email. Zhang, who has not been forthcoming about his role, told us he was not familiar with the manuscript. I have never seen it, he told us in October.

The version of the twins manuscript we have is missing two critically important disclosures usually present in scientific papers. First, it gives no information about who funded the project or what financial interests the authors have in the outcome. Also missing is a section in which each authors scientific contribution is detailed. This means the text does not explicitly describe the role of the single non-Chinese author, Michael Deem of Rice University in Texas. The nature of Deems roleparticularly any hands-on involvement with the patientscould determine penalties that Deem, or his university, could face. Deems lawyers did not answer questions, including a request for copies of his past statements, which sought to minimize his role in the research. Rice says its investigation is ongoing.

13. The researchers ignored evidence that the gene edits werent uniform

In data attached to the paper, in the so-called supplementary material, are tables that He previously showed publicly. It shows chromatograms, or the readout of the DNA sequences found in the embryos and birth tissues of the twins (the umbilical cord and placenta) when his team tried to measure what editing had happened to the CCR5 gene.

Some observers, including Musunuru in our accompanying op-ed, say these data show clearly that the embryos are mosaic, meaning that different cells in the embryo were edited differently. He says presence of multiple edits is visible in the chromatograms, where several distinct readings are registered in overlapping signals at a given DNA position.

The implication of the data is that the twins bodies could be composites of cells edited in different ways, or not at all. That, Musunuru points out, means only some of their cells might have the HIV-resistant gene edit; it also means some might have undetected "off-target" edits, which could potentially cause health problems. The problem of mosaicism was well known to He from his experiments on animal embryos. One of the mysteries of the research project is why He chose to proceed with embryos if they were flawed in this way.

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In his manuscript, He doesnt resolve the mystery. It says only, The CCR5 gene was deep sequenced for all samples to examine the mosaicism of gene editing. Theres no interpretation of what was found, and no acknowledgement that the data seem to show mosaicism or that its a problem.

Fyodor Urnov: They should have worked and worked and worked until they reduced mosaicism to as close to zero as possible. This failed completely. They forged ahead anyway.

See the original post:
Chinas CRISPR babies: Read exclusive excerpts from the unseen original research - MIT Technology Review

Its been a year since He Jiankui announced that hed made the worlds first gene-edited human babies, twin girls with the pseudonyms Lulu and Nana. Widespread condemnation of his actions followed the announcement. But the facts of the case remain unclear, because he has not been transparent about his work.

In his single public appearance following his announcement, at the Second International Summit on Human Genome Editing in Hong Kong in November 2018, He presented his work by racing through about 60 slides in just 20 minutes. Although he showed data about what he had done to the twins genes, it was blink-and-youll-miss-it, and not enough to convince anyone of his claim that hed safely edited the genomes of the human IVF embryos that became Lulu and Nana.

At the summit, He did say hed just submitted a manuscript describing this work to a scientific journal. Twelve months later, however, the manuscript has remained unpublished and its contents mysterious.

He was asked at the summit why he hadnt posted his manuscript to a preprint server such as bioRxiv or on a public websitesomething scientists frequently do to invite feedback on early drafts. He claimed that hed intended to do so, but colleagues had advised him to allow the manuscript to go through peer review by other scientists before posting it. (Normally, formal peer review takes place only when an academic journal is considering publishing a paper.)

By deciding not to release his manuscript right away, He has made it difficult for other scientists to figure out exactly what he did and how he did it. We already know that there were profound ethical problems with Hes work in germline gene editing, which refers to genetic alterations to embryosor to egg or sperm cellsthat can be passed down through the generations. But its scientific merit, and especially its safety, have remained in question.

When I first had the opportunity to look through a complete manuscript from He last November, I immediately realized there were problems.

The most serious was rampant mosaicism. This means that the gene edits He made to the embryos didnt take effect uniformly: different cells showed different changes. Evidence of mosaicism is present in both Lulus and Nanas embryos, as well as in Lulus placenta, making it likely the twins themselves are mosaic. Some parts of their bodies may contain the specific edits He said he made, other parts may contain other edits he didnt highlight, and yet other parts may contain no edits at all. This would mean that the purported benefit of Hes editing HIV resistancemay not extend to the twins entire bodies, and they could still be fully vulnerable to HIV.

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When judging whether the embryos had edits, He took a few cells from the 200 to 300 present in an IVF embryo and analyzed their DNA. But it was the remaining cells went on to multiply to make up the full body. So it is possible that some parts of the twins bodies have edits that He didnt intend to make (off-target edits) and never had a chance to see. Such off-target edits could cause problems such as cancer and heart disease, and could be passed on to Lulus and Nanas future children.

He apparently didnt realize that his own data revealed extensive mosaicism in the embryos, since he made no note of it in the manuscript I saw. Some have wondered if the CRISPR twins were actually a hoax, but to me, the flaws evident in the data make it clear that they werent. Rather, Hes work was a graphic demonstration of attempted gene editing gone awry. Two living human beings, and potentially their descendants too, will bear the consequences.

You shouldnt have to take my word for any of this. You should be able to judge for yourself, or at least hear what other scientists have to say about it.

However, it seems increasingly unlikely that He will be publishing in a peer-reviewed journal. For one thing, I doubt that any respectable journal would seriously consider publishing research with such ethical problems. And even if one did, and sent the manuscript for peer review, He would be in no position to respond to any technical criticisms with further experimental work. He has been under house arrest, and his laboratory was shut down shortly after his announcement about the twins last year.

The only reason to continue keeping Hes work under wraps would be to preserve his ability to publish it someday in a peer-reviewed journal and earn the imprimatur of scientific quality. The community is under no obligation to grant him this privilege. Indeed, it owes him no professional courtesy at all, any more than it would have owed such courtesy to the doctors responsible for the medical experiments in Nazi Germany or the American scientists in charge of the Tuskegee syphilis study.

Rather, in light of the egregious scientific and ethical lapses inherent in Hes cavalier and secretive efforts to make the worlds first gene-edited babies, it is he who owes all of us a full accounting of his actions. Since he has shirked his responsibility to make his work public, its up to others to step in.

Why must the information be public? Its because Hes work reveals serious, unresolved safety concerns. Its not clear that any effort to directly edit human embryos, even if done ethically and with full social approval, can reliably avoid these problems.

International committees convened by the World Health Organization, the US National Academies of Medicine and Sciences, and the Royal Society are currently working to propose regulatory frameworks for doing clinical germline gene editing safely, if it is to be done at all. How can the committees properly do their work without fully understanding all the scientific problems with the single real-world application of clinical germline gene editing thats been attempted to date?

Most worrying is that scientists like Denis Rebrikov in Russia aspire to follow in Hes footsteps. Rebrikov has said hell be able to edit the human germline safely. But how can Rebrikov credibly claim to be able to do better than He if the nature of the problems with Hes work arent widely known? How can the Russian authorities properly evaluate the safety of his proposals without being able to refer to Hes work for guidance?

Its time for the scientific community to fully understand what happened with Lulu and Nana, and to avoid stumbling down a path toward further ill-starred experiments with clinical germline gene editing.

Kiran Musunuru is an associate professor of cardiovascular medicine and genetics at the Perelman School of Medicine at the University of Pennsylvania and the author of The CRISPR Generation, a book about the history of gene editing and the Chinese twins.

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Opinion: We need to know what happened to CRISPR twins Lulu and Nana - MIT Technology Review