Archive for the ‘Crispr’ Category

CRISPR may have generated a lot of buzz this year, but some researchers are already looking beyond it to the next new gene-editing technique. Say hello to prime editing.

If CRISPR is like scissors then you can think of prime editors like word processors, said chemist David Liu in an October press briefing. He spoke days ahead of the first-ever prime editing study, published in Nature and authored by Liu and his team at the Broad Institute of MIT and Harvard University. Liu explained that, while CRISPR cuts through DNAs double helix to snip out genes, prime editing searches for and replaces targeted genes without such slicing and dicing, reducing the risk of unintended changes to the genetic code.

The team was able to correct mutations associated with both sickle cell and Tay-Sachs diseases. Liu believes the technique ultimately might be able to correct almost 90 percent of such mutations, but stressed additional studies are needed to gauge prime editings full potential.

This is the beginning, rather than the end, said Liu.

[This story appeared in print as "It's Prime Time for a New Gene Editor."]

Read this article:
Will Prime Editors be the New CRISPR? - Discover Magazine

First we will be looking for the boiling points and excitability of CRISPR Therapeutics AG (CRSP) stock, it purposes common trait for traders and value investors.

Volatility Indicators for CRISPR Therapeutics AG:

Volatility of the CRISPR Therapeutics AG remained at 5.00% over last week and shows 4.74% volatility in last month. In addition to number of shares traded in last few trading sessions volatility also tells about the fluctuation level of the stock price, commonly a high volatility is the friend of day traders. Volatility is also measured by ATR an exponential moving average (14-days) of the True Ranges. Currently, the ATR value of companys stock is situated at 3.33.

CRISPR Therapeutics AG (CRSP) traded 1718001 shares in most recent trading session as compared to an average volume of 1.06M shares. It shows that the shares were traded in the recent trading session and traders shown interest in CRSP stock. Listed Shares of the CRISPR Therapeutics AG (CRSP) moved down -2.60% to trade at $59.32 on Thursday (01/02/2020) trading session. It has a market capitalization of $3.41B. Knowing about the market capitalization of a company helps investor to determine the company size, market value and the risk. The stock EPS is $-0.46 against its recent stock value of $59.32 per share.

Now entering into the performance part of the article on CRISPR Therapeutics AG stock we should check the stocks actual performance in the past.

Performance of the CRSP Stock:

CRISPR Therapeutics AG revealed performance of -11.77% during the period of last 5 trading days and shown last 12 months performance of 98.59%. The stock moved to 23.84% in last six months and it maintained for the month at -13.55%. The stock noted year to date 2019 performance at -2.60% and changed about 51.75% over the last three months. The stock is now standing at -19.84% from 52 week-high and is situated at 118.46% above from 52-week low price.

Technical Indicators of CRISPR Therapeutics AG Stock:

RSI momentum oscillator is the most common technical indicator of a stock to determine about the momentum of the shares price and whether the stock trading at normal range or its becoming oversold or overbought. It also helps to measure Speed and change of stock price movement. RSI reading varies between 0 and 100. Commonly when RSI goes below 30 then stock is oversold and stock is overbought when it goes above 70. So as currently the Relative Strength Index (RSI-14) reading of CRISPR Therapeutics AG stock is 39.32.

Although it is important to look for trades in a direction of bigger trends when stocks are indicating an opposite short-term movement. Like looking for overbought conditions when bigger trend remained down and oversold conditions when bigger trend is up. In order to check a bigger trend for CRSP a 14-day RSI can fell short and considered as a short-term indicator. So in that situation a Simple moving average of a stock can also be an important element to look in addition to RSI.

The share price of CRSP is currently down -11.59% from its 20 days moving average and trading 0.16% above the 50 days moving average. The stock price has been seen performing along overhead drift from its 200 days moving average with 26.32%. Moving averages are an important analytical tool used to identify current price trends and the potential for a change in an established trend. The simplest form of using a simple moving average in analysis is using it to quickly identify if a security is in an uptrend or downtrend.

Profitability Spotlight for CRISPR Therapeutics AG:

Operating Margin which tells about what proportion of a companys revenue is left over after paying for variable costs of production such as wages & raw materials is noted at -4.50%. Net profit margin of the company is -5.30% that shows how much the company is actually earning by every dollar of sales.

Return on Investment (ROI) of stock is -40.70%. ROI ratio tells about the efficiency of a number of investments in a company. Return on Assets (ROA) which shows how much the company is profitable as compared to its total assets is observed at -2.00%. Return on Equity (ROE), which tells about the profitability of the corporation by evaluating the profit it generates in ratio to the money shareholders have invested, is noted at -2.60%.

Analysts Estimation on Stock:

The current analyst consensus rating stood at 2.1 on shares (where according to data provided by FINVIZ, 1.0 Strong Buy, 2.0 Buy, 3.0 Hold, 4.0 Sell, 5.0 Strong Sell). Analysts opinion is also an important factor to conclude a stocks trend. Many individual analysts and firms give their ratings on a stock. While Looking ahead of 52-week period, the mean Target Price set by analysts is $77.5.

See the original post here:
What to Analyze About the CRISPR Therapeutics AG (CRSP)? - News Welcome

From finding the building blocks for life on Mars to breakthroughs in gene editing and the rise of artificial intelligence, here are six major scientific discoveries that shaped the 2010s - and what leading experts say could come next

We don't yet know whether there was ever life on Mars - but thanks to a small, six-wheeled robot, we do know the Red Planet was habitable.

Shortly after landing on 6 August 2012, NASA's Curiosity rover discovered rounded pebbles - new evidence that rivers flowed there billions of years ago.

The proof has since multiplied, showing there was in fact a lot of water on Mars - the surface was covered in hot springs, lakes, and maybe even oceans.

A crater on the Red Planet filled with water ice. (ESA/DLR/FU Berlin, CC BY-SA 3.0 IGO)

Curiosity also discovered what NASA calls the building blocks of life, complex organic molecules, in 2014.

And so the hunt continues for signs that Earth-based life is not (or wasn't always) alone.

Two new rovers will be launched next year - America's Mars 2020 and Europe's Rosalind Franklin rovers, looking for ancient microbes.

"Going into the coming decade, Mars research will shift from the question 'Was Mars habitable?' to 'Did (or does) Mars support life?'" said Emily Lakdawalla, a geologist at The Planetary Society.

We had long thought of the little corner of the Universe that we call home as unique, but observations made thanks to the Kepler space telescope blew apart those pretensions.

Launched in 2009, the Kepler mission helped identify more than 2,600 planets outside of our Solar System, also known as exoplanets - and astronomers believe each star has a planet, meaning there are billions out there.

Kepler's successor TESS was launched by NASA in 2018, as we scope out the potential for extraterrestrial life.

Expect more detailed analysis of the chemical composition of these planets' atmospheres in the 2020s, said Tim Swindle, an astrophysicist at the University of Arizona.

We also got our first glimpse of a black hole this year thanks to the groundbreaking work of the Event Horizon Telescope collaboration.

(Event Horizon Telescope Collaboration)

"What I predict is that by the end of the next decade, we will be making high quality real-time movies of black holes that reveal not just how they look, but how they act on the cosmic stage," Shep Doeleman, the project's director, told AFP.

But one event from the decade undoubtedly stood above the rest: the detection for the first time on September 14, 2015 of gravitational waves, ripples in the fabric of the universe.

The collision of two black holes 1.3 billion years earlier was so powerful it spread waves throughout the cosmos that bend space and travel at the speed of light. That morning, they finally reached Earth.

The phenomenon had been predicted by Albert Einstein in his theory of relativity, and here was proof he was right all along.

Three Americans won the Nobel prize in physics in 2017 for their work on the project, and there have been many more gravitational waves detected since.

Cosmologists meanwhile continue to debate the origin and composition of the universe. The invisible dark matter that makes up its vast majority remains one of the greatest puzzles to solve.

"We're dying to know what it might be," said cosmologist James Peebles, who won this year's Nobel prize in physics.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) - a family of DNA sequences - is a phrase that doesn't exactly roll off the tongue.

(Meletios Verras/iStock)

But the field of biomedicine can now be divided into two eras, one defined during the past decade: before and after CRISPR-Cas9 (or CRISPR for short), the basis for a gene editing technology.

"CRISPR-based gene editing stands above all the others," William Kaelin, a 2019 Nobel prize winner for medicine, told AFP.

In 2012, Emmanuelle Charpentier and Jennifer Doudna reported that they had developed the new tool that exploits the immune defense system of bacteria to edit the genes of other organisms.

It is much simpler than preceding technology, cheaper and easy to use in small labs.

Charpentier and Doudna were showered in awards. but the technique is also far from perfect and can create unintended mutations.

Experts believe this may have happened to Chinese twins born in 2018 as a result of edits performed by a researcher who was widely criticized for ignoring scientific and ethical norms.

Still, CRISPR remains one of the biggest science stories of recent years, with Kaelin predicting an "explosion" in its use to combat human disease.

For decades, doctors had three main weapons to fight cancer: surgery, chemotherapy drugs, and radiation.

The 2010s saw the rise of a fourth, one that was long doubted: immunotherapy, or leveraging the body's own immune system to target tumor cells.

(Design Cells/iStock)

One of the most advanced techniques is known as CAR T-cell therapy, in which a patient's T-cells - part of their immune system - are collected from their blood, modified and reinfused into the body.

A wave of drugs have hit the market since the mid-2010s for more and more types of cancer including melanomas, lymphomas, leukemias and lung cancers - heralding what some oncologists hope could be a golden era.

For William Cance, scientific director of the American Cancer Society, the next decade could bring new immunotherapies that are "better and cheaper" than what we have now.

The decade began with a major new addition to the human family tree: Denisovans, named after the Denisova Cave in the Altai Mountains of Siberia.

Scientists sequenced the DNA of a female juvenile's finger bone in 2010, finding it was distinct both from genetically modern humans and Neanderthals, our most famous ancient cousins who lived alongside us until around 40,000 years ago.

The mysterious hominin species is thought to have ranged from Siberia to Indonesia, but the only remains have been found in the Altai region and Tibet.

We also learned that, unlike previously assumed, Homo sapiens bred extensively with Neanderthals - and our relatives were not the brutish simpletons previously assumed but were responsible for artworks, such as the handprints in a Spanish cave they were credited for crafting in 2018.

They also wore jewelry, and buried their dead with flowers - just like we do.

Next came Homo naledi, remains of which were discovered in South Africa in 2015, while this year, paleontologists classified yet another species found in the Philippines: a small-sized hominin called Homo luzonensis.

Advances in DNA testing have led to a revolution in our ability to sequence genetic material tens of thousands of years old, helping unravel ancient migrations, like that of the Bronze Age herders who left the steppes 5,000 years ago, spreading Indo-European languages to Europe and Asia.

"This discovery has led to a revolution in our ability to study human evolution and how we came to be in a way never possible before," said Vagheesh Narasimhan, a geneticist at Harvard Medical School.

One exciting new avenue for the next decade is paleoproteomics, which allows scientists to analyze bones millions of years old.

"Using this technique, it will be possible to sort out many fossils whose evolutionary position is unclear," said Aida Gomez-Robles, an anthropologist at University College London.

"Neo" skull of Homo naledi from the Lesedi Chamber. (John Hawks/University of the Witwatersrand)

Machine learning - what we most commonly mean when talking about "artificial intelligence" - came into its own in the 2010s.

Using statistics to identify patterns in vast datasets, machine learning today powers everything from voice assistants to recommendations on Netflix and Facebook.

So-called "deep learning" takes this process even further and begins to mimic some of the complexity of a human brain.

It is the technology behind some of the most eye-catching breakthroughs of the decade: from Google's AlphaGo, which beat the world champion of the fiendishly difficult game Go in 2017, to the advent of real-time voice translations and advanced facial recognition on Facebook.

In 2016, for example, Google Translate - launched a decade earlier - transformed from a service that provided results that were stilted at best, nonsensical at worst, to one that offered translations that were far more natural and accurate.

At times, the results even seemed polished.

"Certainly the biggest breakthrough in the 2010s was deep learning - the discovery that artificial neural networks could be scaled up to many real-world tasks," said Henry Kautz, a computer science professor at the University of Rochester.

"In applied research, I think AI has the potential to power new methods for scientific discovery," from enhancing the strength of materials to discovering new drugs and even making breakthroughs in physics, Kautz said.

For Max Jaderberg, a research scientist at DeepMind, owned by Google's parent company Alphabet, the next big leap will come via "algorithms that can learn to discover information, and rapidly adapt and internalize and act on this new knowledge," as opposed to depending on humans to feed them the correct data.

That could eventually pave the way to "artificial general intelligence", or a machine capable of performing any tasks humans can, rather than excelling at a single function.

Agence France-Presse

Original post:
These 6 Incredible Discoveries From The Past Decade Have Changed Science Forever - ScienceAlert

Often, science moves ahead incrementally. Yet sometimes it advances in dramatic leaps and bounds that can stir new hopes for medical benefits while shaking society to its very core. We saw both in 2019, as work using the gene-editing tool CRISPR gained momentum.

The year opened with the scientific community scrambling to respond to the news that Chinese researcher He Jiankui had used CRISPR technology to edit the genomes of human embryos. The experiments resulted in the November 2018 birth of twin girls, the first humans with genetically altered germline cells which means their genetic changes are heritable and can be passed on to future generations.

The National Institutes of Health (NIH), a crucial public supporter of biomedical research, is among the many who consider such experiments both irresponsible and unethical. The NIH has not funded any use of heritable gene-editing technologies in human embryos, and it has no intention of doing so.

But NIHs strong stance against heritable gene editing does not mean that we think CRISPR has no role in the future of clinical medicine. This innovative technology possesses enormous potential for therapeutic good if used for making nonheritable genetic changes. In that approach, genetic material is altered only in relevant somatic (nonreproductive) cells, so there is no chance of passing those changes on to future offspring.

NIHs highest priorities in 2019 included supporting research aimed at using nonheritable gene editing to help people with life-threatening disorders, such as sickle cell disease, HIV infection, cancer and muscular dystrophy. Indeed, such applications may offer the best hope not only for treating, but for curing, many of the nearly 6,000 human genetic diseases that still lack treatments.

Now, scientists and leaders around the globe have an obligation to consider the appropriate use if any of heritable human gene editing. This involves scrutinizing the safety of such experiments, including the risk of unintended mutations, as well as a clear-eyed analysis of actual medical need. In our view, the current arguments that the benefits outweigh the risks are surprisingly uncompelling. But our deliberations should not stop there. We must weigh the profound social, ethical and moral issues associated with modifying the germline in ways that could change the human species forever.

Given the significance of these decisions, in March, leading scientists from seven countries led by Eric Lander and including CRISPR pioneers Feng Zhang and Emmanuelle Charpentier called for a five-year international moratorium on the use of gene editing to modify the human germline for clinical purposes. The NIH supports such a moratorium.

Despite the calls for caution, some researchers are forging ahead. In June, Russian molecular biologist Denis Rebrikov announced plans to implant gene-edited embryos into women. Like his Chinese counterpart, Rebrikov planned to use CRISPR to target the CCR5 gene to protect against HIV; he later changed course to focus on GJB2, a gene linked to heritable hearing loss.

Direct editing of embryos is not the only way to alter the human germline in heritable ways: In August, New York-based reproductive biologist Gianpiero Palermo went public with his plans to use CRISPR technology to target a gene that increases cancer risk in human sperm.

But such moves continued to meet vigorous opposition. In August, a number of research groups working on gene-editing therapeutics issued a statement asserting heritable gene editing is currently inappropriate for use in human clinical studies. That same month, a group of international research societies convened to discuss recommendations for appropriate research, which are slated for completion in spring of 2020.

Meanwhile, the World Health Organizations new expert advisory committee, convened in the wake of Hes experiments, sidestepped the issue of a moratorium at its August meeting. It did, however, establish a global registry to track all kinds of human gene-editing research and to offer consultation on governance of such technologies.

A moratorium of at least five years on heritable human gene editing would provide us time to engage in proactive, rather than reactive, discussions about the future of such technology. That discussion has to be inclusive of many societal perspectives. We must never allow our technology to eclipse our humanity. As an interconnected global society, we have a responsibility to ask ourselves some very hard questions about heritable gene editing and the dangers of human hubris. While difficult, this is a debate that we simply cannot afford to postpone.

Francis S. Collins is the director of the National Institutes of Health.

Read this article:
NIH Director on Human Gene Editing: 'We Must Never Allow our Technology to Eclipse our Humanity' - Discover Magazine

NewsScienceThis was the decade designer babies went from concept to feasibility

Saturday, 28th December 2019, 7:02 am

Gene editing

This was the decade when designer babies went from science fiction to fact as a Chinese scientist, He Jiankui, made the shock announcement in December 2018 that the worlds first genetically modified children had been born. He was working illegally and he was widely condemned for not waiting until regulations had been put into place.

But the move showed just how rapidly the Crispr-Cas9 gene-editing technique likened to a find and replace command wasadvancing.

Embryonic and pluripotent Stem Cellresearch

This potentially revolutionary field of medicine has developed to the point where treatments are just around the corner.

Embryonic, or pluripotent, stem cells have extraordinary medical potential because they can develop into any one of the 220 or so mature, specialised cells of the body from insulin-making pancreatic cells to the nerve cells of the brain. In 2018, scientists restored the vision of two UK patients with age-related macular degeneration by inserting a patch of embryonic stem cells into their eyes. The research team hopes an affordable, off-the-shelf therapy could be available to NHS patients within five years.

Treatments for spinal cord injury, heart failure, diabetes, Parkinsons disease and lung cancer are also in advanced trials.

Higgs Boson

Gravitational waves

Scientific history was made in December 2016 as gravitational ripples in the fabric of spacetime, first predicted by Albert Einstein 100 years earlier, were detected, opening new vistas into the dark side of the universe. Physicists around the world confirmed they had detected unambiguous signals of gravitational waves emanating from the collision of two black holes 1.3 billion light years away.

The observations not only confirmed Einsteins general theory of relativity; they also provided the first direct detection of black holes colliding.

Black holes

Neanderthals

The Neanderthals may have been extinct for thousands of years, but in 2010, geneticists mapped their genome using DNA extracted from ancient bones. This led to a startling discovery: our ancestors interbred with other species after they migrated out of Africa.

So in the UK, most of us have a small percentage of Neanderthal genes in our DNA.

Read more:
From gene editing to black holes and the Neanderthals, here's the biggest advances in science over the past decade - inews

National Science Foundation via Getty Images

December 28, 2019

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Capturing a black holeScientists this year unveiled the first-ever image of a black hole. One of the universe's most enigmatic phenomena, a black hole is a cosmic abyss so dense with matter that not even light can escape. The picture from Messier 87, a galaxy 55 million light-years away, shows a bright ring of particles heated to billions of degrees, circling a supermassive black hole some 25 billion miles wide. To capture the image, scientists focused eight ultrapowerful radio telescopes around the world on Messier 87 for 10 days. Researchers compared the resulting images to millions of simulations of what the black hole might look like and found a match. "We have seen what we thought was unseeable," says project leader Shep Doeleman.

Fighting EbolaEbola could soon be classified as a curable disease, thanks to a trial of experimental treatments in Congo. An epidemic of the hemorrhagic disease has killed more than 2,200 people in the African nation; about 70 percent of those who catch Ebola die. But the death rate plummeted to 29 percent for patients on one new drug, and 34 percent for those on another. Among patients who began treatment soon after developing symptoms, rates fell to 6 percent and 11 percent, respectively. The drugs use monoclonal antibodies: Y-shaped proteins that call on immune cells to attack the Ebola virus.

The new CRISPRScientists have developed a gene-editing tool that could one day correct 89 percent of the genetic mutations that cause inherited diseases, such as cystic fibrosis and sickle cell anemia. The most popular existing gene-editing approach, CRISPR-Cas9, uses "molecular scissors" to locate faulty genetic code, then cuts both strands of the DNA double helix and splices in a new section of code. Though cheap and fast, this process often damages nearby code or inserts the new material in the wrong place. The new technology, known as prime editing, cuts only one strand of the double helix minimizing the risk of unintended changes.

Mind-reading robo-suitA quadriplegic man in France can move all four of his paralyzed limbs again, thanks to a pioneering brain-controlled bodysuit. The patient, identified only as Thibault, had two implants surgically placed over the parts of his brain that control movement. His brain signals are sent to a nearby computer, which translates them into movement instructions for the exoskeleton suit. Though years away from being publicly available, the technology could pave the way for mind-controlled wheelchairs and similar equipment. Thibault said taking his first steps felt like being "the first man on the moon."

Previously on The Week: The 34 most memorable looks of the 2010s

Blood test for Alzheimer'sDoctors could soon use a simple blood test to predict if a patient will develop Alzheimer's, years before symptoms appear. An international team of researchers found that people genetically predisposed to the disorder had distinctly higher levels of neurofilament light chain a protein found in the brain and spinal cord seven years before symptoms began, and noticeably faster-growing levels more than 16 years in advance. An early test could help scientists determine whether new Alzheimer's drugs are effective. Study author Mathias Jucker says the reason there is no effective treatment for the condition is "partly because current therapies start much too late."

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The biggest scientific breakthroughs of 2019 - The Week

A gene editing technology which utilises special proteins found in the natural defence mechanisms of bacteria which can chop up DNA of invading organisms and destroy them. CRISPR (pronounced "crisper" and standing for "Clustered Regularly Interspaced Short Palindromic Repeats") can quickly and relatively cheaply provide alterations to DNA sequences and correct genetic defects causing diseases ranging from cystic fibrosis to cataracts.

3D printing

This decade has seen 3D printing become more accessible, cheaper and efficient. The manufacturing system allows designers and engineers to perfectly transfer a digital model to a physical three-dimensional part. Accurate models can be created by putting down material a layer at a time which also facilitates intricate and complex structures. This means anything can be created such as tools, boats and even topological maps without additional assembly.

SpaceX reusable launch system

A method that enables space-going rockets to take-off and land without jettisoning any elements of the craft. By maintaining a single reusable structure, SpaceX can drastically minimise the costs of space exploration. Traditional rockets need to release not only the giant external tank but also its solid rocket boosters. Replacing these components can be lengthy and expensive. The technology to land a rocket vertically is very impressive and a must-see.

Power banks

Energy storage devices have the capacity to change the world. Historically it has been difficult to be completely self-sufficient on clean and renewable energies as systems because solar power is reliant on the sun and that source is lost at night. Devices such as Tesla's power wall offer a way to cut emissions for the average household.

Cloud computing

Cloud computing products think Microsoft Azure and Amazon Web Services allow companies and individuals to access and administer complex services without the hassle of personally maintaining underlying hardware and software. The upshot? We can all enjoy office productivity suites without having any onsite servers.

Read more from the original source:
Technology: Sean Dimmock's top five innovations and advances of the decade - HeraldScotland

The pancreas is a multi-tasker. Unfortunately, so is its most common cancer.

A pancreatic cancer diagnosis can carry the weight of a death sentence more than nine out of every 10 people dont live to see the 5 year anniversary of their diagnosis. Celebrity diagnoses, like Alex Trebek's, popularize an anxiety felt by the roughly 50,000 people diagnosed each year. The disease progresses so quickly, andfrequently without detection, that treatment is often futile. But, new research reveals how pancreatic cancer cells spread with ease and how we might stop their hostile takeover.

The multitasking pancreas.

Max Levy

Shaped like a comma and nestled atop the small intestine, the pancreas is responsible for a vital arsenal of hormones that help maintain blood sugar levels. But it also spends time working with its downstairs neighbor, the intestine, to help us digest. It produces a thick juice, (actual technical term) that pours down a duct to supply the small intestine with enzymes that break down carbs, fats, and proteins. Cancer of that duct, pancreatic ductal adenocarcinoma, claims over 40,000 lives every year. By the time a doctor breaks the news to their patient, that cancer has likely spread elsewhere and will be difficult to treat.

Most cancers are riddled with avenues of perforated blood vessels. Ambitious cancer cells can use them to spread tumors elsewhere, but those avenues also make it easier to treat the disease with drugs. With this type of pancreatic duct cancer, its the worst of both worlds.

Adenocarcinoma of the pancreas. The dark purple circles in the center of the tumor are the nuclei of cancer cells.

Ed Uthman on Flickr

They escape really early but they dont have a lot of vessels, says Duc-Huy Nguyen, a co-author of the study. A healthy pancreas has far more blood vessels than a cancerous one. According to Nguyen, some doctors have reported diseased pancreases with structures that look likenormal blood vessels, but are in fact nonfunctional tubes lined with cancer cells rather than healthy ones. Nobody knew why or how the cancer could kill and replace healthy cells.

Nguyen knew that the answer likely had to do with a biological competition between different types of cells: cancerous pancreatic cells, and healthy endothelial cells that line nearby bloodstreams.

You have two different cell types and you place them next to one another, says Nguyen, If you ask who is going to win? Id believe the tumor cell is going to.

Nguyen and his team designed a clear plastic chip to support pancreatic cancer cells beside an artificial bloodstream lined with endothelial cells. With total visibility and control, the team could watch cancer cells inch towards their rivals. Nguyen expected to see a typical process called intravasation when cancer cells casually slip past healthy ones to enter the bloodstream. He was surprised to witness something far more destructive.

Alex Trebek's recent diagnosis of pancreatic cancer brought the condition into the limelight

Anders Krusberg/Peabody Awards via Wikimedia Commons

The cancer cells muscle their way into a stronghold over their neighbors. First, they sneak up on the endothelial cells; then they wrap around them like sleeve. Endothelial cells represent a sort of blockade, preventing undesired cells from entering the bloodstream. But this mob of cancer cells isaggressive, and quick to overpower this blockade meant to keep them out of the blood. Before long, this protective barrier of endothelial cells is no more, and cancerous cells fill the channel. The cancer can then juggle a lethal combination of offensive and defensive maneuvers: hijacking blood vessels to isolate itself from attack, and dispatchingeven more cancer cells to spread elsewhere.

Zooming in on the drama was interesting to Nguyen, but the science could leap from interesting to useful ifhe could devise a way to slow the cancer's progression.So we just asked the question: what really happens there? Nguyen and his team set out toeavesdrop on the chemical conversations going on between cells.

They began by tinkering with the cancers main line of communication with the outside world: a horde of chemicals and receptors called the transforming growth factor beta (TGF-) signaling pathway. Finding the right chemical signal that prompts the cancer's outward progression, Nguyen says, could reveal a treatment that stops the takeover in its tracks.

The team quickly confirmed they were on to something. Using a TGF--inhibiting drug in their the chip-model, Nguyen switched off large chunks of the signaling pathway as soon as the cancer reached healthy cells. That change significantly reduced the blood vessel destruction.

Encouraged, the team used CRISPR gene editing to delete individual receptors in both cell types. One by one, Nguyen examined the cancers progress, hoping to pinpoint a secret weapon. He finally found that weapon in the form of a receptor called ALK7. By knocking out the pancreatic cancer cells ALK7, they rescued the endothelial cells from certain defeat. The team then repeated the experiment using mice carrying the genetically modified cancer and found that mouse blood vessels survived far better than those exposed to the unmodified cancer. Hampering that receptor amounted to swapping out the cancers bazooka for a rubber pistol.

I thought this was important work, says Melissa Skala, a cancer researcher at the Morgridge Institute not affiliated with the study. There are very few treatment options for pancreatic cancer, so any development that enables more targeted drugs is very important.

I felt they were actually very careful with their claims, says Shannon Stott, a scientist at Massachusetts General Hospital unaffiliated with this study. Stott creates devices to study how cancer spreads and paid keen attention to the design of the cancer model. She says this experiment has an important balance: it is simple enough to study the disease reliably, and complex enough to obtain potentially critical for real patients. Its a clever, elegant study.

Since cancer is such a diverse disease, this treatment cant treat every type of cancer. But Nguyen believes that some, such as ovarian cancer, may respond to the same treatment.

Were still far from using this work to treat patients. Still, models like this one give us a platform to understand diseases and pinpoint their weaknesses. My takeaway was not, Oh, we can start treating patients with this, says Stott. It opens the door for us to expand upon this to ask interesting questions.

Read the rest here:
To stop pancreatic cancer from spreading, cut out the chatter - Massive Science

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States. Meredith Rizzo/NPR hide caption

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States.

When Victoria Gray was just 3 months old, her family discovered something was terribly wrong.

"My grandma was giving me a bath, and I was crying. So they took me to the emergency room to get me checked out," Gray says. "That's when they found out that I was having my first crisis."

It was Gray's first sickle cell crisis. These episodes are one of the worst things about sickle cell disease, a common and often devastating genetic blood disorder. People with the condition regularly suffer sudden, excruciating bouts of pain.

"Sometimes it feels like lightning strikes in my chest and real sharp pains all over. And it's a deep pain. I can't touch it and make it better," says Gray. "Sometimes, I will be just balled up and crying, not able to do anything for myself.

Gray is now 34 and lives in Forest, Miss. She volunteered to become the first patient in the United States with a genetic disease to get treated with the revolutionary gene-editing technique known as CRISPR.

NPR got exclusive access to chronicle Gray's journey through this medical experiment, which is being watched closely for some of the first hints that changing a person's genes with CRISPR could provide a powerful new way to treat many diseases.

"This is both enormously exciting for sickle cell disease and for all those other conditions that are next in line," says Dr. Francis Collins, director of the National Institutes of Health.

"To be able to take this new technology and give people a chance for a new life is a dream come true," Collins says. "And here we are."

Doctors removed bone marrow cells from Gray's body, edited a gene inside them with CRISPR and infused the modified cells back into her system this summer. And it appears the cells are doing what scientists hoped producing a protein that could alleviate the worst complications of sickle cell.

"We are very, very excited," says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating Gray.

Frangoul and others stress that it's far too soon to reach any definitive conclusions. Gray and many other patients will have to be treated and followed for much longer to know whether the gene-edited cells are helping.

"We have to be cautious. It's too early to celebrate," Frangoul says. "But we are very encouraged so far."

Collins agrees.

"That first person is an absolute groundbreaker. She's out on the frontier," Collins says. "Victoria deserves a lot of credit for her courage in being that person. All of us are watching with great anticipation."

This is the story of Gray's journey through the landmark attempt to use the most sophisticated genetic technology in what could be the dawn of a new era in medicine.

The study took place at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe. Meredith Rizzo/NPR hide caption

The study took place at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe.

Life filled with pain

When I first meet her, Gray is in a bed at the TriStar Centennial Medical Center in Nashville wearing a hospital gown, big gold hoop hearings and her signature glittery eye shadow.

It's July 22, 2019, and Gray has been in the hospital for almost two months. She is still recovering from the procedure, parts of which were grueling.

Nevertheless, Gray sits up as visitors enter her room.

"Nice to meet y'all," she says.

Gray is just days away from her birthday, which she'll be celebrating far from her husband, her four children and the rest of her family. Only her father is with her in Nashville.

"It's the right time to get healed," says Gray.

Gray describes what life has been like with sickle cell, which afflicts millions of people around the world, including about 100,000 in the United States. Many are African American.

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company. Meredith Rizzo/NPR hide caption

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company.

"It's horrible," Gray says. "When you can't walk or, you know, lift up a spoon to feed yourself, it gets real hard."

The disease is caused by a genetic defect that turns healthy, plump and pliable red blood cells into deformed, sickle-shaped cells. The defective cells don't carry oxygen well, are hard and sticky and tend to clog up the bloodstream. The blockages and lack of oxygen wreak havoc in the body, damaging vital organs and other parts of the body.

Growing up, Victoria never got to play like other kids. Her sickle cells made her weak and prone to infections. She spent a lot of time in the hospital, recovering, getting blood transfusions all the while trying to keep up with school.

"I didn't feel normal. I couldn't do the regular things that every other kid could do. So I had to be labeled as the sick one."

Gray made it to college. But she eventually had to drop out and give up her dream of becoming a nurse. She got a job selling makeup instead but had to quit that too.

The sickle-shaped cells eventually damaged Gray's heart and other parts of her body. Gray knows that many patients with sickle cell don't live beyond middle age.

"It's horrible knowing that I could have a stroke or a heart attack at any time because I have these cells in me that are misshapen," she says. "Who wouldn't worry?"

Gray says she understands the risks involved in the treatment. "This gives me hope if it gives me nothing else," she says. Meredith Rizzo/NPR hide caption

Gray says she understands the risks involved in the treatment. "This gives me hope if it gives me nothing else," she says.

Gray married and had children. But she hasn't been able to do a lot of things most parents can, like jump on a trampoline or take her kids to sporting events. She has often had to leave them in the middle of the night to rush to the hospital for help.

"It's scary. And it affected my oldest son, you know, because he's older. So he understands. He started acting out in school. And his teacher told me, 'I believe Jemarius is acting out because he really believes you're going to die,' " Gray says, choking back tears.

Some patients can get help from drugs, and some undergo bone marrow transplants. But that procedure is risky; there's no cure for most patients.

"It was just my religion that kind of kept me going," Gray says.

An eager volunteer

Gray had been exploring the possibility of getting a bone marrow transplant when Frangoul told her about a plan to study gene editing with CRISPR to try to treat sickle cell for the first time. She jumped at the chance to volunteer.

"I was excited," Gray says.

CRISPR enables scientists to edit genes much more easily than ever before. Doctors hope it will give them a powerful new way to fight cancer, AIDS, heart disease and a long list of genetic afflictions.

"CRISPR technology has a lot of potential use in the future," Frangoul says.

To try to treat Gray's sickle cell, doctors started by removing bone marrow cells from her blood last spring.

Next, scientists used CRISPR to edit a gene in the cells to turn on the production of fetal hemoglobin. It's a protein that fetuses make in the womb to get oxygen from their mothers' blood.

"Once a baby is born, a switch will flip on. It's a gene that tells the ... bone marrow cells that produce red cells to stop making fetal hemoglobin," says Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's TriStar Centennial Medical Center.

The hope is that restoring production of fetal hemoglobin will compensate for the defective adult-hemoglobin sickle cells that patients produce.

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells don't carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain. Ed Reschke/Getty Images hide caption

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells don't carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain.

"We are trying to introduce enough ... fetal hemoglobin into the red blood cell to make the red blood cell go back to being happy and squishy and not sticky and hard, so it can go deliver oxygen where it's supposed to," Frangoul says.

Then on July 2, after extracting Gray's cells and sending them to a lab to get edited, Frangoul infused more than 2 billion of the edited cells into her body.

"They had the cells in a big syringe. And when it went in, my heart rate shot up real high. And it kind of made it hard to breath," Gray says. "So that was a little scary, tough moment for me."

After that moment passed, Gray says, she cried. But her tears were "happy tears," she adds.

"It was amazing and just kind of overwhelming," she says, "after all that I had went through, to finally get what I came for."

The cells won't cure sickle cell. But the hope is that the fetal hemoglobin will prevent many of the disease's complications.

"This opens the door for many patients to potentially be treated and to have their disease modified to become mild," Frangoul says.

The procedure was not easy. It involved going through many of the same steps as a standard bone marrow transplant, including getting chemotherapy to make room in the bone marrow for the gene-edited cells. The chemotherapy left Gray weak and struggling with complications, including painful mouth sores that made it difficult to eat and drink.

But Gray says the ordeal will have been worth it if the treatment works.

She calls her new gene-edited cells her "supercells."

"They gotta be super to do great things in my body and to help me be better and help me have more time with my kids and my family," she says.

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer. Meredith Rizzo/NPR hide caption

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer.

Concerns about risk

Other doctors and scientists are excited about the research. But they're cautious too.

"This is an exciting moment in medicine," says Laurie Zoloth, a bioethicist at the University of Chicago. "Everyone agrees with that. CRISPR promises the capacity to alter the human genome and to begin to directly address genetic diseases."

Still, Zoloth worries that the latest wave of genetic studies, including the CRISPR sickle cell study, may not have gotten enough scrutiny by objective experts.

"This a brand-new technology. It seems to work really well in animals and really well in culture dishes," she says. "It's completely unknown how it works in actual human beings. So there are a lot of unknowns. It might make you sicker."

Zoloth is especially concerned because the research involves African Americans, who have been mistreated in past medical studies.

Frangoul acknowledges that there are risks with experimental treatments. But he says the research is going very slowly with close oversight by the Food and Drug Administration and others.

"We are very cautious about how we do this trial in a very systematic way to monitor the patients carefully for any complications related to the therapy," Frangoul says.

Gray says she understands the risks of being the first patient and that the study could be just a first step that benefits only other patients, years from now. But she can't help but hope it works for her.

Dr. Haydar Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, is leading the study in Nashville. Meredith Rizzo/NPR hide caption

Dr. Haydar Frangoul, medical director of pediatric hematology/oncology at HCA Healthcare's Sarah Cannon Research Institute and TriStar Centennial Medical Center, is leading the study in Nashville.

She imagines a day when she may "wake up and not be in pain" and "be tired because I've done something not just tired for no reason." Perhaps she could play more with her kids, she says, and look forward to watching them grow up.

"That means the world to me," Gray says.

It could be many weeks or even months before the first clues emerge about whether the edited cells are safe and might be working.

"This gives me hope if it gives me nothing else," she says in July.

Heading home at last

About two months later, Gray has recovered enough to leave the hospital. She has been living in a nearby apartment for several weeks.

Enough time has passed since Gray received the cells for any concerns about immediate side effects from the cells to have largely passed. And her gene-edited cells have started working well enough for her immune system to have resumed functioning.

So Gray is packing. She will finally go home to see her children in Mississippi for the first time in months. Gray's husband is there to drive her home.

"I'm excited," she says. "I know it's going to be emotional for me. I miss the hugs and the kisses and just everything."

After living for months in Nashville, where the study was taking place, Gray packs her bags to finally go home to her kids and family in Forest, Miss. Meredith Rizzo/NPR hide caption

After living for months in Nashville, where the study was taking place, Gray packs her bags to finally go home to her kids and family in Forest, Miss.

Gray is wearing bright red glittery eye shadow. It matches her red tank top, which repeats "I am important" across the front.

She unzips a suitcase and starts pulling clothes from the closet.

"My goodness. Did I really bring all this?" she says with a laugh.

Before Gray can finish packing and depart, she has to stop by the hospital again.

"Are you excited about seeing the kids?" Frangoul says as he greets her. "Are they going to have a big welcome sign for you in Mississippi?"

Turns out that Gray has decided to make her homecoming a surprise.

"I'm just going to show up tomorrow. Like, 'Mama's home,' " she says, and laughs.

After examining Gray, Frangoul tells her that she will need to come back to Nashville once a month for checkups and blood tests to see if her genetically modified cells are producing fetal hemoglobin and giving her healthier red blood cells.

"We are very hopeful that this will work for Victoria, but we don't know that yet," Frangoul says.

Gray will also keep detailed diaries about her health, including how much pain she's experiencing, how much pain medication she needs and whether she needs any blood transfusions.

"Victoria is a pioneer in this. And we are very excited. This is a big moment for Victoria and for this pivotal trial," Frangoul says. "If we can show that this therapy is safe and effective, it can potentially change the lives of many patients."

Gray hopes so too.

See more here:
Sickle Cell Therapy With CRISPR Gene Editing Shows Promise : Shots - Health News - NPR

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.

Picture: Supplied.

WASHINGTON, United States - 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 email.

"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 cofounder 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 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 $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 the 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 ex-communication 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."

See the original post here:
2019: The year gene therapy came of age - Eyewitness News

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States.

Meredith Rizzo/NPR

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Meredith Rizzo/NPR

Victoria Gray, who has sickle cell disease, volunteered for one of the most anticipated medical experiments in decades: the first attempt to use the gene-editing technique CRISPR to treat a genetic disorder in the United States.

Meredith Rizzo/NPR

When Victoria Gray was just 3 months old, her family discovered something was terribly wrong.

My grandma was giving me a bath, and I was crying. So they took me to the emergency room to get me checked out, Gray says. Thats when they found out that I was having my first crisis.

It was Grays first sickle cell crisis. These episodes are one of the worst things about sickle cell disease, a common and often devastating genetic blood disorder. People with the condition regularly suffer sudden, excruciating bouts of pain.

Sometimes it feels like lightning strikes in my chest and real sharp pains all over. And its a deep pain. I cant touch it and make it better, says Gray. Sometimes, I will be just balled up and crying, not able to do anything for myself.

Gray is now 34 and lives in Forest, Miss. She volunteered to become the first patient in the United States with a genetic disease to get treated with the revolutionary gene-editing technique known as CRISPR.

NPR got exclusive access to chronicle Grays journey through this medical experiment, which is being watched closely for some of the first hints that changing a persons genes with CRISPR could provide a powerful new way to treat many diseases.

This is both enormously exciting for sickle cell disease and for all those other conditions that are next in line, says Dr. Francis Collins, director of the National Institutes of Health.

To be able to take this new technology and give people a chance for a new life is a dream come true, Collins says. And here we are.

Doctors removed bone marrow cells from Grays body, edited a gene inside them with CRISPR and infused the modified cells back into her system this summer. And it appears the cells are doing what scientists hoped producing a protein that could alleviate the worst complications of sickle cell.

We are very, very excited, says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tenn., who is treating Gray.

Frangoul and others stress that its far too soon to reach any definitive conclusions. Gray and many other patients will have to be treated and followed for much longer to know whether the gene-edited cells are helping.

We have to be cautious. Its too early to celebrate, Frangoul says. But we are very encouraged so far.

Collins agrees.

That first person is an absolute groundbreaker. Shes out on the frontier, Collins says. Victoria deserves a lot of credit for her courage in being that person. All of us are watching with great anticipation.

This is the story of Grays journey through the landmark attempt to use the most sophisticated genetic technology in what could be the dawn of a new era in medicine.

The study took place at HCA Healthcares Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe.

Meredith Rizzo/NPR

hide caption

toggle caption

Meredith Rizzo/NPR

The study took place at HCA Healthcares Sarah Cannon Research Institute and TriStar Centennial Medical Center, in Nashville, Tenn., one of 11 sites recruiting patients for the research in the U.S., Canada and Europe.

Meredith Rizzo/NPR

Life filled with pain

When I first meet her, Gray is in a bed at the TriStar Centennial Medical Center in Nashville wearing a hospital gown, big gold hoop hearings and her signature glittery eye shadow.

Its July 22, 2019, and Gray has been in the hospital for almost two months. She is still recovering from the procedure, parts of which were grueling.

Nevertheless, Gray sits up as visitors enter her room.

Nice to meet yall, she says.

Gray is just days away from her birthday, which shell be celebrating far from her husband, her four children and the rest of her family. Only her father is with her in Nashville.

Its the right time to get healed, says Gray.

Gray describes what life has been like with sickle cell, which afflicts millions of people around the world, including about 100,000 in the United States. Many are African American.

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company.

Meredith Rizzo/NPR

hide caption

toggle caption

Meredith Rizzo/NPR

In July, Gray was recovering after a medical procedure that infused billions of her own bone marrow cells back into her body after they had been modified using the gene-editing technique CRISPR. Her father, Timothy Wright (right), traveled from Mississippi to keep her company.

Meredith Rizzo/NPR

Its horrible, Gray says. When you cant walk or, you know, lift up a spoon to feed yourself, it gets real hard.

The disease is caused by a genetic defect that turns healthy, plump and pliable red blood cells into deformed, sickle-shaped cells. The defective cells dont carry oxygen well, are hard and sticky and tend to clog up the bloodstream. The blockages and lack of oxygen wreak havoc in the body, damaging vital organs and other parts of the body.

Growing up, Victoria never got to play like other kids. Her sickle cells made her weak and prone to infections. She spent a lot of time in the hospital, recovering, getting blood transfusions all the while trying to keep up with school.

I didnt feel normal. I couldnt do the regular things that every other kid could do. So I had to be labeled as the sick one.

Gray made it to college. But she eventually had to drop out and give up her dream of becoming a nurse. She got a job selling makeup instead but had to quit that too.

The sickle-shaped cells eventually damaged Grays heart and other parts of her body. Gray knows that many patients with sickle cell dont live beyond middle age.

Its horrible knowing that I could have a stroke or a heart attack at any time because I have these cells in me that are misshapen, she says. Who wouldnt worry?

Gray says she understands the risks involved in the treatment. This gives me hope if it gives me nothing else, she says.

Meredith Rizzo/NPR

hide caption

toggle caption

Meredith Rizzo/NPR

Gray says she understands the risks involved in the treatment. This gives me hope if it gives me nothing else, she says.

Meredith Rizzo/NPR

Gray married and had children. But she hasnt been able to do a lot of things most parents can, like jump on a trampoline or take her kids to sporting events. She has often had to leave them in the middle of the night to rush to the hospital for help.

Its scary. And it affected my oldest son, you know, because hes older. So he understands. He started acting out in school. And his teacher told me, I believe Jemarius is acting out because he really believes youre going to die, Gray says, choking back tears.

Some patients can get help from drugs, and some undergo bone marrow transplants. But that procedure is risky; theres no cure for most patients.

It was just my religion that kind of kept me going, Gray says.

An eager volunteer

Gray had been exploring the possibility of getting a bone marrow transplant when Frangoul told her about a plan to study gene editing with CRISPR to try to treat sickle cell for the first time. She jumped at the chance to volunteer.

I was excited, Gray says.

CRISPR enables scientists to edit genes much more easily than ever before. Doctors hope it will give them a powerful new way to fight cancer, AIDS, heart disease and a long list of genetic afflictions.

CRISPR technology has a lot of potential use in the future, Frangoul says.

To try to treat Grays sickle cell, doctors started by removing bone marrow cells from her blood last spring.

Next, scientists used CRISPR to edit a gene in the cells to turn on the production of fetal hemoglobin. Its a protein that fetuses make in the womb to get oxygen from their mothers blood.

Once a baby is born, a switch will flip on. Its a gene that tells the bone marrow cells that produce red cells to stop making fetal hemoglobin, says Frangoul, medical director of pediatric hematology/oncology at HCA Healthcares TriStar Centennial Medical Center.

The hope is that restoring production of fetal hemoglobin will compensate for the defective adult-hemoglobin sickle cells that patients produce.

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells dont carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain.

Ed Reschke/Getty Images

hide caption

toggle caption

Ed Reschke/Getty Images

Patients with sickle cell disease have blood cells that are stiff and misshapen. The cells dont carry oxygen as well and clog up the bloodstream, resulting in periodic bouts of excruciating pain.

Ed Reschke/Getty Images

We are trying to introduce enough fetal hemoglobin into the red blood cell to make the red blood cell go back to being happy and squishy and not sticky and hard, so it can go deliver oxygen where its supposed to, Frangoul says.

Then on July 2, after extracting Grays cells and sending them to a lab to get edited, Frangoul infused more than 2 billion of the edited cells into her body.

They had the cells in a big syringe. And when it went in, my heart rate shot up real high. And it kind of made it hard to breath, Gray says. So that was a little scary, tough moment for me.

After that moment passed, Gray says, she cried. But her tears were happy tears, she adds.

It was amazing and just kind of overwhelming, she says, after all that I had went through, to finally get what I came for.

The cells wont cure sickle cell. But the hope is that the fetal hemoglobin will prevent many of the diseases complications.

This opens the door for many patients to potentially be treated and to have their disease modified to become mild, Frangoul says.

The procedure was not easy. It involved going through many of the same steps as a standard bone marrow transplant, including getting chemotherapy to make room in the bone marrow for the gene-edited cells. The chemotherapy left Gray weak and struggling with complications, including painful mouth sores that made it difficult to eat and drink.

But Gray says the ordeal will have been worth it if the treatment works.

She calls her new gene-edited cells her supercells.

They gotta be super to do great things in my body and to help me be better and help me have more time with my kids and my family, she says.

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer.

Meredith Rizzo/NPR

hide caption

toggle caption

Meredith Rizzo/NPR

Gray was diagnosed with sickle cell disease as an infant. She was considering a bone marrow transplant when she heard about the CRISPR study and jumped at the chance to volunteer.

Meredith Rizzo/NPR

Concerns about risk

Other doctors and scientists are excited about the research. But theyre cautious too.

This is an exciting moment in medicine, says Laurie Zoloth, a bioethicist at the University of Chicago. Everyone agrees with that. CRISPR promises the capacity to alter the human genome and to begin to directly address genetic diseases.

Still, Zoloth worries that the latest wave of genetic studies, including the CRISPR sickle cell study, may not have gotten enough scrutiny by objective experts.

Read more:
Sickle Cell Therapy With CRISPR Gene Editing Shows Promise : Shots - Invest Records

Many scientists assume that if a chunk of a gene is missing then the protein that it encodes will not function, or even be produced.

Researchers at the European Molecular Biology Laboratory in Heidelberg, Germany used CRISPR to make cuts in 136 different genes. In about a third of cases, proteins were still produced from these damaged genes and, furthermore, many of the proteins remained partially functional. This strange phenomenon, of damaged DNA producing functional protein, does more than punctuate lifes remarkable adaptability and resilience.

It means that dozens, or hundreds, of studies that used CRISPR/Cas9 to knock out genes, but failed to validate that the encoded protein was fully removed, could be incorrect or misleading.

The problem with major scientific developments, especially CRISPR/Cas9, is that experimental tools often explode in popularity before scientists and editors can implement standard procedures.

Unfortunately, academic institutions and scientific publishers are hulking bureaucracies with slow-moving policy changes. Ensuring that CRISPR/Cas9 produces repeatable experiments rather than blemishes on the scientific record will require the collective action of scientists. It will demand self-governance.

Read full, original post: Heres why many CRISPR/Cas9 experiments could be wrong and how to fixthem

See the original post:
Viewpoint: There's a repeatability problem with CRISPR experiments. Only 'self-governance' can fix it - Genetic Literacy Project

CRISPR, kick-starting the revolution in drug discovery or A year after the first CRISPR babies, stricter regulations are now in place. read some of the recent headlines. CRISPR, a new gene editing technology, is making waves around the world and Israel is no exception. The Israeli startup eggXTt is preparing to use CRISPR-tech to mark chicken eggs by gender in an effort to reduce waste in the poultry industry, and research labs at institutes around the country regularly make use of CRISPR-tech to make groundbreaking discoveries in the biological sciences.

But how does CRISPR actually work, and what are the limitations of this new technology? CRISPR is often touted by scientists and science journalists as a pair of molecular scissors allowing us to edit our genomes at will in a point-and-click fashion. Although it is tempting to believe these buzzwords, they are not particularly accurate, and can be misleading for the public and policymakers considering the potential impacts of this new technology. After all, our DNA is not a tiny Microsoft Word document that can be altered however we see fit. In this article we will dive into exactly what CRISPR is, what it can and cannot do, and why we might not be seeing designer CRISPR babies for a few more decades (or centuries).

First of all, CRISPR is not a pair of molecular scissors. It is a system of proteins that evolved in bacteria to protect them against viruses. Proteins can take all shapes and sizes, and CRISPR proteins look something like the wire cleaning scrubbers you can find in many kitchens. The oft-mentioned analogy that CRISPR are molecular scissors is doubly misleading, because scissors imply that someone (ie: scientists) are somehow wielding them in a precise manner to cut and paste DNA as they please. This gives the false impression that scientists are the sole possessors of CRISPR knowledge, bestowing upon them the power to alter our genomes at will.

In reality, CRISPR proteins slide along DNA strands, recognizing specific areas by their unique feel. More specifically, the proteins move along the DNA until they find a spot on the DNA that matches perfectly with their recognition site, and then they squeeze down and cause the DNA to break at that point. This is similar to how your handprint fits well into its imprint in the sand. When you think about the wide variety of proteins in the human body (over 100,000) it makes sense that few other proteins would make the same match (a rubber duck or iron nail would not fit well into your handprint either). When the CRISPR proteins move along the DNA, they are only able to make the DNA break at these specific points. Scientists are able to take advantage of this tendency of CRISPR proteins, and can manipulate them to make breaks in DNA at the area they want removed or altered in their experiments. The CRISPR system also consists of a few other components, including a set of guide RNAs that help the CRISPR proteins match up with the DNA of their choice.

Unfortunately, CRISPR proteins are not perfect, and DNA is a very long and repetitive molecule, so it is possible for mistakes to occur. Other areas of DNA may look the same to the CRISPR proteins due to similar or identical sequences, causing the CRISPR proteins to break the DNA at undesired places. Recent research has noted that CRISPR can have a high frequency of off-target DNA breaks, up to 50% in many model systems. These issues mean that once CRISPR is released into a living organism it is sometimes hard to predict where these off target effects will occur. The challenge of off-target effects is one of the reasons CRISPR babies are likely a long way off. As a result a number of institutions and many scientists, including the World Health Organization, have called for a comprehensive ban on genetic modifications to reproductive or germline tissues. Despite this, a team of researchers in China recently managed to create a set of genetically altered twins, resulting in significant controversy. The ethical questions surrounding CRISPR in humans are another compelling reason to wait, particularly because edits of germline tissues like eggs and sperm could result in permanent changes to the human genome.

Another issue with the CRISPR system is that it needs to be inserted into living cells using a viral vector. This means the CRISPR system has to be translated into DNA, coded into a type of non-deadly virus, and injected into cells, which then produce the CRISPR proteins themselves. These viral systems are never 100% successful, and sometimes only enter 15-20% of all cells, which is not ideal for medical-grade treatments.

Despite these barriers there are several medical treatments in development using CRISPR-tech to address difficult-to-treat diseases. One of the most advanced is a CRISPR-based treatment for Duchenne Muscular Dystrophy (DMD), a rare and incurable muscle degenerative disease predominantly affecting children. DMD is caused by mutations in the dystrophin gene and is always fatal with an average patient lifespan of 26 years. Recent studies in mouse models and human heart cells in petri dishes have shown that CRISPR can cause reduction in muscular degeneration symptoms, which are the hallmark of this disease. Because DMD is caused by mutations in one specific region in the genome, scientists and clinicians can take advantage of CRISPRs targeted DNA-breakage effects to chop the affected section out of the genome by targeting two RNA guide probes, one to each side of the mutant piece of DNA. In most cases simply excising the mutant piece of DNA is not sufficient to remove symptoms of a disease. However, in this rare case removing the mutant DNA section allows for a partial improvement in some muscle cells, which is why this treatment has shown promise for clinical applications.

Many of the future CRISPR-based treatments will need to insert a new, healthy piece of DNA in addition to removing the mutant DNA. This is obviously many times more difficult as in addition to mitigating risk from off-target CRISPR effects, it will also be necessary to reduce the risk of the new piece of DNA inserting into the wrong portion of the genome and causing undesirable effects. Nevertheless, trials are now underway to translate this treatment method to the clinic in studies investigating the use of CRISPR for Sickle-Cell Anemia, Cystic Fibrosis and non-Hodgkins Lymphoma.

Although the major benefits of CRISPR-tech are likely decades away, CRISPR is already having significant impacts in the scientific, medical and biotech spheres. As long as this technology is used responsibly, we have much to gain from a world where we could one day become the masters of our own genomes.

This is an article in the series Science & Technology in the Holy Land, a regular column on innovations in science, tech, start-ups and futurism by Jamie Magrill, an MSc, Biomedical Sciences Candidate at the Hebrew University of Jerusalem.

Jamie Magrill is a scientist-scholar and world-traveler with an interest in entrepreneurship and startups, particularly in the biomedical and philanthropic fields, an MSc in Biomedical Sciences Candidate at the Hebrew University of Jerusalem, and a Masa Israel Journey alum.

Read the original:
CRISPR: Are we the Masters of our Own Genomes? - The Times of Israel

As the old saying goes, strike when the iron is hot. That's what a new gene editing start-up named Beam Therapeutics hopes to do by conducting an initial public offering (IPO) less than two years after forming and more than a year before it asks regulators for permission to begin clinical trials. Given the excitement over genetic medicines, it might be wise to take advantage of the open window now.

Assuming the IPO proceeds as planned, Beam Therapeutics will offer investors a second chance to own a next-generation gene editing technology platform and the first next-generation CRISPR tool. Here's why investors might want to keep the business on their radar.

Image source: Getty Images.

Beam Therapeutics bears some similarities to Editas Medicine (NASDAQ:EDIT). Both trace their origins back to the Broad Institute in Boston. They share a trio of all-star scientific founders: Dr. Feng Zhang, Dr. David Liu, and Dr. Keith Joung. Each company's technology platform is built on CRISPR-based tools.

But the differences are more important for investors. Editas Medicine is developing gene editing tools that require Cas enzymes to cut both strands of DNA. While that theoretically provides the ability to delete or insert genetic sequences to treat diseases, the approach relies on innate DNA repair mechanisms. When the built-in safeguards on those mechanisms break down, cells can turn cancerous. CRISPR-CasX tools can also create unintended genetic edits, and have a relatively low efficiency.

Beam Therapeutics is developing gene editing tools based on a new technique called base editing. The enzymatic approach doesn't make double-stranded breaks in DNA. Instead, it induces chemical reactions to change the sequence of the genetic alphabet -- A (adenine), T (thymine), C (cytosine), and G (guanine) -- one letter at a time. Base editing can make A-to-G edits, C-to-T edits, G-to-A edits, and T-to-C edits.

The next-generation approach decouples CRISPR gene editing tools and the need to make double-stranded breaks in DNA, which is the most pressing concern facing Editas Medicine, CRISPR Therapeutics (NASDAQ:CRSP), and Intellia Therapeutics (NASDAQ:NTLA).

Clinical Consideration

CRISPR-CasX Gene Editing

CRISPR Base Editing

Does it cut DNA?

Yes, enzymatically cuts both strands of DNA

No

Can be used to insert new genetic material into a sequence?

Yes

No, but it can enzymatically change an existing DNA sequence

Does it trigger DNA repair mechanisms?

Yes

No

Source: Beam Therapeutics, author.

While base editing can't make every possible edit (example: A-to-T edits), it can target a number of disease-driving genetic errors. And Beam Therapeutics has inked important collaboration deals to augment the capabilities of its technology platform:

After reviewing the details, investors see that there's a tangled web of related transactions that all flow back to the Broad Institute, which is going to great lengths to extract every ounce of value from its scientific discoveries. Similar actions have caused a stir in the scientific community in recent years. If the profit-seeking terms of the non-profit research institution's agreements are too strict, then it may pose a risk to Beam Therapeutics at the expense of investors.

Image source: Getty Images.

Investors familiar with gene editing stocks will immediately recognize the programs included in the pipeline of the base editing pioneer. The lead assets take aim at blood disorders, and are part of a push to engineer better immunotherapies to treat cancer.

In beta thalassemia and sickle cell disease, Beam Therapeutics is first attempting to increase the production of fetal hemoglobin, which confers natural immunity to both conditions. That's similar to the lead drug candidate of CRISPR Therapeutics, which recently demonstrated promising results from the first two patients in a phase 1 clinical trial.

A second program in sickle cell disease aims to directly correct the genetic mutation responsible for the blood disorder. It involves changing a single base -- perfectly suited for base editing.

In immunotherapy, Beam Therapeutics is working to engineer better chimeric antigen receptor T (CAR-T) cells that can be used as cellular medicines to treat various types of cancers. CRISPR Therapeutics, Editas Medicine, and Intellia Therapeutics are deploying CRISPR gene editing in the same applications, while Precision BioSciences (NASDAQ:DTIL) is leaning on ARCUS gene editing to do the same. The latter's lead drug candidates are in immunotherapy, a unique distinction among gene editing stocks.

Beam Therapeutics' pipeline also includes a range of potential assets aimed at gene correction, gene silencing, and more complex editing, but none have entered clinical trials. The company doesn't expect to file investigational new drug (IND) applications -- required for regulators to sign off on the start of clinical trials -- until 2021. But since the window for an IPO might be slammed shut by then, the business is exploring a market debut now.

There aren't many details in the company's S1 filing concerning a potential date for a market debut or how much money the company is aiming to raise. The filing says $100 million, but that's just a placeholder for the initial submission. The actual amount will be determined once Wall Street gets an idea of the level of interest in an IPO, which will determine the number of shares to offer and the price.

Assuming the IPO takes place, Beam Therapeutics and base editing offer investors a technological upgrade over the first-generation gene editing platforms leaning on CRISPR-CasX tools. The next-generation tools aren't perfect, and there are risks related to the agreements with the Broad Institute and sister start-ups, but this is certainly a gene editing stock worth watching.

More here:
This Start-up Might Be the Next Gene Editing IPO - The Motley Fool

The most you can lose on any stock (assuming you don't use leverage) is 100% of your money. But when you pick a company that is really flourishing, you can make more than 100%. For instance the CRISPR Therapeutics AG (NASDAQ:CRSP) share price is 212% higher than it was three years ago. Most would be happy with that. On top of that, the share price is up 35% in about a quarter.

View our latest analysis for CRISPR Therapeutics

Because CRISPR Therapeutics made a loss in the last twelve months, we think the market is probably more focussed on revenue and revenue growth, at least for now. When a company doesn't make profits, we'd generally expect to see good revenue growth. Some companies are willing to postpone profitability to grow revenue faster, but in that case one does expect good top-line growth.

CRISPR Therapeutics's revenue trended up 87% each year over three years. That's much better than most loss-making companies. Along the way, the share price gained 46% per year, a solid pop by our standards. This suggests the market has recognized the progress the business has made, at least to a significant degree. Nonetheless, we'd say CRISPR Therapeutics is still worth investigating - successful businesses can often keep growing for long periods.

You can see below how earnings and revenue have changed over time (discover the exact values by clicking on the image).

NasdaqGM:CRSP Income Statement, December 20th 2019

CRISPR Therapeutics is a well known stock, with plenty of analyst coverage, suggesting some visibility into future growth. So we recommend checking out this free report showing consensus forecasts

We're pleased to report that CRISPR Therapeutics rewarded shareholders with a total shareholder return of 187% over the last year. That's better than the annualized TSR of 46% over the last three years. The improving returns to shareholders suggests the stock is becoming more popular with time. You could get a better understanding of CRISPR Therapeutics's growth by checking out this more detailed historical graph of earnings, revenue and cash flow.

If you are like me, then you will not want to miss this free list of growing companies that insiders are buying.

Please note, the market returns quoted in this article reflect the market weighted average returns of stocks that currently trade on US exchanges.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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If You Had Bought CRISPR Therapeutics (NASDAQ:CRSP) Stock Three Years Ago, You Could Pocket A 212% Gain Today - Yahoo Finance

Every living cell on Earth has proteins. Typically thousands of them, that serve as molecular machines to digest food, sense the environment, or anything else a cell must do. However, many genes, and the proteins they code for, have unknown functions. In humans, the function of about 1 out of 5 of genes is unknown. To explore these dark corners of the genome, scientists can break up DNA to disable a gene, making their encoded proteins nonfunctional, and watch what happens to cells as a result, inferring the lost function from what goes wrong.

When CRISPR/Cas9 came online in 2012, it offered scientists a tool to do exactly this cut genes. The Cas9 enzyme searches through DNA, using a guide RNA to look for a specific sequence, and makes a cut when it finds a match. The gene, split in two, is repaired by the cell, but often with a large chunk missing. Many scientists assume that if a chunk of a gene is missing then the protein that it encodes will not function, or even be produced.

In many cases, they would be terriblywrong.

Researchers at the European Molecular Biology Laboratory in Heidelberg, Germany used CRISPR to make cuts in 136 different genes. In about a third of cases, proteins were still produced from these damaged genes and, furthermore, many of the proteins remained partially functional. This strange phenomenon, of damaged DNA producing functional protein, does more than punctuate lifes remarkable adaptability and resilience.

It means that dozens, or hundreds, of studies that used CRISPR/Cas9 to knock out genes, but failed to validate that the encoded protein was fully removed, could be incorrect or misleading.

Also read: How Gene Editing Is Changing the World

While many labs that use CRISPR to knock out genes dovalidate that the encoded protein is no longer produced, other labs fail to check. Searching for one protein in a cell is time-consuming and technically challenging; testing for protein function takes even longer. There are some methodsavailableto look for specific proteins, but many CRISPR/Cas9 studies fail to run these experiments or scientific journals dont ask for the data.

Nature Methods, the same journal that published the paper from the Heidelberg laboratory, recognised shortcomings in CRISPR validation early on. In 2017, they highlighted a genome-editing consortium, in collaboration with theUS National Institute of Standards and Technology, that aims to develop standardized procedures for CRISPR research, including publishing guidelines that include which guide RNAs were tested, how they were designed, and which controls were used in experiments.

The problem with major scientific developments, especially CRISPR/Cas9, is that experimental tools often explode in popularity before scientists and editors can implement standard procedures. When DNA sequencing was developed in the 1970s, for instance, there was little need for standards because it was so challenging to decipher the sequence of even a short piece of DNA. A decade later, however, GenBank, a DNA sequence repository, came online and journals began to mandate that researchers deposit their sequences. This requirement, together with reporting standards issued by journals likeNature, have ensured that a rapidly growing collection of DNA sequences can be vetted and independently analysed by the scientific community. The same is true for methods like x-ray crystallography, with journals requiring that protein structures be independently validated and uploaded to publicly-accessible databases.

Today, every laboratory uses CRISPR/Cas9 in a different way, reports their findings in a different journal, and checks their results with a different technique. There is often little uniformity, and that needs tochange.

But while some scientists were shocked by the new study, others took a laissez-faire approach to the findings. On Twitter, many vented their rage at what they felt was a lack of careful controls by the scientific community. Raphael Ferreira, a postdoctoral fellow at Harvard Medical School, was inspired, perceiving this study as a game changer for the CRISPR community.

I was as surprised by the results in a really positive way, as this paper rings the wake-up call for every scientist using CRISPR/Cas9, Ferreira said.

Despite the enthusiasm, however, Ferreira will not change how he performs his own experiments. The few times I have [used CRISPR/Cas9] in mammalian cells, I have always confirmed them with a Western blot, referring to an experimental method to detect specific proteins.

Victor de Lorenzo, a research professor at the National Center for Biotechnology in Madrid, agreed, claiming that, the only way to ensure that a protein is altogether removed is by making a Western blot.

Down the hall or across the street from my office, dozens of scientists use CRISPR/Cas9 to uncover protein functions. One of these researchers is Shashank Gandhi, a PhD student at the California Institute of Technology (CalTech) that has published CRISPR/Cas9 methods to delete genes in chicken embryos. Though he agrees with Ferreira and de Lorenzo, Gandhi asserts that validations could be taken a step further, and believes that journal editors should take action.

Also read: Is There More to Gene Editing Than Creating Designer Humans?

I think that journal editors should encourage authors to present supplemental data on how the knockouts were validated, insists Gandhi. I know that Nature requires that information as part of a research summary document that is submitted to the journal with each paper submission.

If Nature, which is widely considered the premier academic research journal, takes action to ensure that CRISPR knockouts are validated, then perhaps other publishers will take notice. In the meantime, Gandhi and others are not taking any chances.

I use several approaches to validate my CRISPR knockouts. For starters, I design and test multiple [guide RNAs] targeting the same gene for all my knockout experiments. Secondly, wherever applicable, I try to perform rescue experiments to establish loss of function phenotypes, says Gandhi, referring to an experiment in which a deleted gene is restored to test whether that proteins phenotype returns, confirming a link between a gene and the function that was lost when the gene was broken.

While all of the scientists that I spoke with agreed that researchers could do more to double check their experiments, it is unclear what actionable steps could be taken. Perhaps a combination of scientific, academic, and institutional changes could alleviate the potential for misleading studies. Faster experimental methods to detect proteins, standardised publishing procedures, and an academic database that describes which guide RNAs have been tested in each organism, would all serve to enhance the rigour of current studies.

Unfortunately, academic institutions and scientific publishers are hulking bureaucracies with slow-moving policy changes. Ensuring that CRISPR/Cas9 produces repeatable experiments rather than blemishes on the scientific record will require the collective action of scientists. It will demand self-governance.

Nicholas McCarty, Bioengineering, California Institute of Technology

This article was published in MassiveScience. Read the original here.

More:
Heres Why Many CRISPR/Cas9 Experiments Could Be Wrong and How to Fix Them - The Wire

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CRISPR And CRISPR-Associated (Cas) Genes Market Growth Analysis By Manufacturers, Regions, Type And Application, Forecast (2019-2026) - Market...

1. New Horizons nails the most distant flyby in history

Just 33 minutes after ringing in the New Year, scientists at the Johns Hopkins University Applied Physics Laboratory cheered and threw confetti a second time. The New Horizons spacecraft had just conducted a flyby of a Kuiper Belt object 4 billion miles from Earth. And as the sun rose on January 1, New Horizons beamed back its first close-up images of the 19-mile-long peanut-shaped space rock, officially named 2014 MU69.

Images eventually revealed 2014 MU69 (initially nicknamed Ultima Thule), to be a surprisingly flat contact binary, a body composed of two once-separated rocks that slowly gravitated toward each other until they lightly touched and fused. Scientists believe the flyby data could offer insight into how planets formed in our solar system billions of years ago.

In November, NASA changed the rocks nickname from Ultima Thule, a term with links to the Nazi party, to Arrokoth, which means sky in the Powhatan/Algonquian language.

2014 MU69 is revealed to be a two-tiered snowman. According to the New Horizons team, this image supports the idea that planets in our system formed as bits of raw planetary matter coalesced over time. Image Credit: NASA/Johns Hopkins University Applied Physics Laboratory/Southwest Research Institute

Twelve years after a first patient was declared to be rid of HIV, another person achieved a similar milestone this year. In March, with the help of a stem cell transplant from a virus-resistant donor, the anonymous individual entered long-term remission from HIV.

In both cases, remission followed a transplant of bone marrow from a person with a mutation in the gene that encodes the protein CCR5, which many HIV strains use to infiltrate cells. Neither treatment was originally intended to eliminate the infection itself, but to treat blood cancers that had spread in both individuals.

While the intervention is likely to be effective in only a small fraction of HIV-positive individuals, the 2019 case shows that its efficacy was more than a one-time event.

In April, we were able to feast our eyes on the first-ever image of a black hole. The black hole, whose image was generated from data captured by a network of eight radio observatories that make up the Event Horizon Telescope, dwells in the center of a galaxy some 55 million light-years away from Earth.

As popular as the black hole image was, another aspect of the story that quickly unfolded online: the contribution of 29-year-old MIT scientist Katie Bouman, who crafted an algorithm to help translate the telescope data into the black hole image. Bouman, captured in a photo with a laptop, beaming behind her folded hands, quickly became a symbol for womens accomplishment in astronomy and computer science. But, Marina Koren writes for The Atlantic, This one image tapped into a multitude of questions about the role of women in science, the myth of the lone genius, and the pressure scientists have to promote themselves and their work on social media.

No one algorithm or person made this image, Bouman later wrote, referring to the black hole picture, in a Facebook post. It required the amazing talent of a team of scientists from around the globe and years of hard work to develop the instrument, data processing, imaging methods, and analysis techniques that were necessary to pull off this seemingly impossible feat.

CRISPR-Cas9 is a tool that lets scientists cut and insert small pieces of DNA at precise areas along a DNA strand. | Photo credit: Public Domain

In April, researchers began the first CRISPR-Cas9 gene-editing clinical trials in people in the United States. In the trials, scientists used CRISPR, a powerful gene-editing technique derived from an ancient bacterial immune system, to combat cancer and blood disorders. That month, two cancer patientsone with myeloma and one with sarcomawere treated using CRISPR.

In the cancer and blood disorder trials, researchers remove some cells from a patients body, edit the cells DNA using CRISPR, and inject the cells back in, now hopefully armed to fight disease, Tina Hesman Saey writes for Science News. But in another trial, conducted by Editas Medicine in Cambridge, Mass., researchers are using CRISPR to edit DNA directly in the human body by snipping a small piece of DNA out of cells in the eyes of people with an inherited form of blindness, Saey writes.

The trials come on the heels of Chinese scientist He Jiankuis gene-editing on twin girls born in November 2018, which was widely criticized as premature and highly unethical. .

Mammal fossils like this one, discovered by a team of paleontologists and paleobotanists led by Tyler Lyson in Corral Bluffs, Colorado, fill in a missing piece of the timeline of life. Image Credit: HHMI Tangled Bank Studios

This year, scientists gleaned new insight into the day the dinosaur-killing asteroid crashed into Earth 66 million years ago, and the first million years after the impact.

In April, paleontologist and graduate student Robert DePalma claimed to unveil an unprecedented time capsule of the asteroid-induced catastrophe. He reported finding scorched tree trunks and hundreds of well-preserved fossil fish beneath sediment at a site in North Dakota, forming a snapshot of the first minutes and hours after impact. (Some experts remain cautious about the finding, due in part to the fact that DePalmas discovery was first announced in a New Yorker article before publication of the peer-reviewed paper.)

Then, in October, new fossils that capture the million-year timeline of life after the dinosaurs died were revealed. Discovered in Colorados Corral Bluffs by paleontologist Tyler Lyson and his team, the fossils showcase the extraordinary resilience of life in the wake of disaster and help reveal the evolutionary journey of the mammals that survived the asteroid.

With the use of artificial intelligence on the rise, one serious flaw continued to make headlines in 2019: racial bias. In October, researchers announced that a particular algorithm, which predicts who might benefit from follow-up care and affects 100 million Americans, underestimates black patients need for additional treatment. The algorithm underestimates the health needs of black patients even when theyre sicker than their white counterparts.

Additionally, the U.S. remains one of the most dangerous developed nations in which to be pregnant and give birth, particularly for minorities. Pregnancy-related deaths are rising in the United States and the main risk factor is being black, Mike Stobbe and Marilynn Marchione write for AP News. A CDC report concludes black women, along with Native Americans and Alaska natives, are three times more likely to die before, during or after having a baby, and more than half of these deaths are preventable, Stobbe and Marchione write.

Also this year, researchers further investigated why black scientists are less likely to receive funding from the National Institutes of Health (NIH) than their white counterparts. A study published in October illustrated that topic choice contributes to the lower rates of NIH awards going to black scientists. Specifically, Jeffrey Mervis writes for Science Magazine, black applicants are more likely to propose approaches, such as community interventions, and topics, such as health disparities, adolescent health, and fertility, that receive less competitive scores from reviewers.

A replica of a fragment of a Denisovan finger found in Denisova Cave, Siberia, in 2008. Image Credit: Thilo Parg, Wikimedia Commons

New findings in 2019 added to anthropologists understanding of Denisovans, a species of early human that likely shared the planet with Homo sapiens as recently as 50,000 years ago.

This fall, scientists learned that although Denisovans DNA ties them more closely to Neanderthals, their fingers may have looked more like ours, suggesting Neanderthals broader digits evolved after their lineage split off from the Denisovans around 410,000 years ago. A few more fossils, Bruce Bower writes for Science News, plus genetic analyses indicated Denisovans were close relatives and occasional mating partners of Neanderthals and Homo sapiens tens of thousands of years ago. But there was too little evidence to say what Denisovans looked like or how they behaved.

Physicists reached a milestone in quantum computing this year, a method of computing that uses quantum physics to solve complex problems quickly.

In October, Google said it had achieved quantum supremacy. Its AI Quantum Team presented evidence that it had built a quantum computer that needs only 200 seconds to solve a problem that would have taken IBMs Summit, the worlds most powerful supercomputer, 10,000 years to crack. Though IBM disputed the claim, others in the computing community are tentatively optimistic about the breakthroughs promise. If validated, it may bring us closer to a future of ultra-efficient computing.

Just when you thought Saturn couldnt get any more awesome, it secured yet another claim to fame: the most known moons of any planet in our solar system (sorry, Jupiter).

On October 7, the International Astronomical Unions Minor Planet Center announced that researchers discovered an additional 20 moons orbiting Saturn, bringing its grand total to a whopping 82. Jupiter, the largest and oldest planet in our solar system, has 79.

The latest discoveries were made possible by Hawaiis Subaru telescope. A team led by Carnegie Sciences Scott S. Sheppard first eyed them in the spring of 2017, but because faraway moons are dim and tough to spot, researchers used Subaru to scan the skies periodically throughout the following years to confirm their finding. Then, they used a computer algorithm to link the data through time and confirm that the moons were indeed reliably orbiting Saturn.

Less than a week after the U.N. climate talks came to a close in Madrid this month, Australia recorded its hottest day ever, one day after its previous record. Just a few months ago, wildfires raged across not only the American West and Australian Outback but also Europe and the Amazon, an occurrence that many climatologists believe may have been exacerbated by climate change-induced drought and high temperatures. And in May, a United Nations report claimed that one million plant and animal species are on the verge of extinctionmore than in any other period in human historywith alarming implications for human survival. The warming climate, which heightens the effects of overfishing, pesticide use, pollution, and urban expansion is a major driver, the report concludes.

Three weeks ago, a bleak climate report, also from the U.N., predicted that global carbon emissions will climb despite promises from almost 200 nations to address climate change, propelling temperatures upward and threatening to shatter the threshold of 2C that scientists say would invite dramatic changes to ecology and the economy, Nathaniel Gronewald writes for Science Magazine. And many declared this months COP25 climate talks to be a massive failure.

But climate activists, particularly teens, have seized the spotlight this year. Greta Thunberg, a 16-year-old Swedish climate activist, was just named TIMEs Person of the Year. And at COP25, official youth constituency representatives expressed their disappointment to leaders and officials, Kartik Chandramouli writes for Mongabay. Do you want to be remembered as the ones who had the chance to act but decided not to as betrayers of our generation, of indigenous people and communities desperately fighting on the ground? Youth representatives said. We are rising, we are fighting and we will win.

Read the rest here:
The top 10 science stories of 2019 | NOVA - NOVA Next

Science and technology are the great drivers of innovation in the worldaround us.Technological and scientific breakthroughs help people every day, bringing drinking water to the needy, access to information through the internet to remote villages, cures for obscure diseases.

Many aspects of scientific discovery are under no ethical questions. But there are alsoa number of scientific endeavors that push the ethical lines of what science should revolve around. While all the areas of controversy we'll look into have great benefits, they also come with a lot of ethical burdens, like harm to animals, people, or the environment.

It all should make us stop and think, at what point do the negatives of innovation overshadow the good that it brings. And is there ever an innovation so beneficial to the world and mankind that it would be worth ethical tragedy on the road to scientific and technological progress? Ponder these questions as we look into 7 ethically controversial areas of science and technology...

Artificial intelligence is at the forefront of techno-jargon these days. Every company that has anything to do with technology is using it as a buzzword to sell their product. "New dog collar with built-in AI to detect when your dog is in distress! Install our simple computer plug-in and we'll optimize your workday."

AI certainlyhas its applications and benefits, but there are areas where it has some extensive drawbacks. Take two key AI technologies that have questionable benefits, or rather extensive drawbacks: deep fakes and Neuralink.

You've probably heard of deep fakes, the face-swapping technology that is used to make world leaders say things they never didor for less family-friendly things.

You might not know about Neuralink though. It's one of Elon Musk's technological endeavors that aims to improve brain-machine interfaces, record memories, and other technological advancements with the brain.

Focusing in on Neuralink first, questions surround the ethics of connecting human brains to machines and utilizing AI to make human brains function better. Ethical questions primarily focus on the development of said technology and potential side-effects. The company's goal is to optimize human brain function, but the testing that will be needed to get there will be extensive. This means human testing, on human brains, with unknown consequences. At what point is the potential promise of drastic technological advancement not worth the potential human loss in the development of the technology?

RELATED: AI CONTINUES TO ACT IN UNPREDICTABLE WAYSSHOULD WE BE CONCERNED?

Moving from Neuralink, we're met with technology, deep fakes, that pose less benefit to humanity. There's arguably little reason that anyone needs to replace someone's face with another's in a videoat least, little reason that isn't nefarious.

Yet, technology exists thanks to artificial intelligence and machine learning. It continues to be researched under the guise of benefits through improved video editing technology, but at the end of the day,there's no way to keep it from being used for negative purposes.

At the end of the day, artificial intelligence has the potential to completely change how we interact with the worldbut are there too many negatives? Time will tell...

Through CRISPR, scientists are able to edit human genomes. That means researchers can alter DNA sequences and alter how our genes function. That means the potential to correct genetic defects, preventing the spread of diseaseorrr for making designer babies.

CRISPR is short for CRISPR-Cas9, a gene-editing tool that utilizes the Cas9 enzyme to cut strands of DNA. It's basicallylike molecular scrapbooking.

The idea and implementation of CRISPR came from how bacteria defend themselves, by chopping up and destroying the DNA of foreign invaders before they are able to take hold of the organism.

CRISPR was just a theory until in 2017, a paper was published demonstrating just how CRISPR worked.

Chinese scientists have started using CRISPR to engineer designer babies or create human babies withedited genes, primarily lacking any tendency towards genetic defects. All of this seems noble and can potentiallyimprove humanity's quality of life, but at what cost? We largely don't know any potential side-effects and if there are any, we're talking about human life.

Designing humans also brings into question what exactly a human is. Are we naturally occurring beings, or does being a human just mean thinking like we do and form or process doesn't matter?

Moving on from human gene editing in CRISPR, we can examine the ethical issues with gene editing on other beings, like plants. Gene editing encompasses anytime a scientist intervenes in an organism's genetics.

This intervention creates GMOs or genetically modified organisms. This results in stronger, more drought-resistant crops. Or crops that have higher yields per acre, among a bounty of other things.

Today, gene editing happens across the world and it is done on both plants and animals, mostly in the pursuit of better food production. Looking into the animal realm,gene editing has been usedto create pigs that aren't susceptible to Porcine Reproductive and Respiratory Syndrom, or PRRS. Gene editing has been used to create pigs that are naturally very resistant to the disease, improving animal welfare.

The gene-editing process for all organisms is overseen by various federal agencies, obviously depending upon the country you're practicing this science in. It raises many ethical concerns, primarily along with the side-effects that might be caused by it, and it is still a much-debated topic by ethicists.

Animal testing is likely the most controversial area of scientific research on this list. Many people couldn't care less while others vehemently oppose it. For years, animal testing has been used to create newer and better pharmaceuticals, better makeup, better shampoos, etc.

The keyword here is "better" as it means better for humans. At the end of the day, animal testing places the prevention of human suffering over the importance of the prevention of animal suffering. In certain cases, the ethical argument for animal testing is easier, i.e. cancer research, or other pursuits that would prevent human death. In other cases, the argument is harder, as the development of a better lipstick.

The ethical debate around animal testing is essentially a real-life trolley problem. On one hand, you have human suffering and on the other, you have animal suffering. And we seem to have no problem with animal suffering as long as it is for a greater cause.

In introducing the subject, we've made it seem fairly cut and dry, but as science goes, it rarely ever is. An increasing number of scientists are starting to question the relevance of continued animal testing at a time where AI and other tech is starting to be able to accurately model and predict biological interfaces. A great deal of animals are harmed in the creation of many of the chemicals, and we musk as ourselves, is it worth it?

The natural progression from animal testing is human testing or trials of medication on human test subjects. Human subject research is often necessary to get drugs to the final phase of regulatory approval. It serves as the final check of how a givenmedicine or chemical will interact with the human system. Yet, time and time again it has hurt, maimed, or killed individuals. And we have to ask ourselves again, at what point does it become not worth it?

History hasn't been kind to the reputation of human trials, though scientists are making aconstant effort to create safety standardsin the process.

In 1947, it was discovered that two German physicians conducted deadly experiments on concentration camp prisoners during WWII. They were prosecuted as war criminals in the Nuremberg Trials. The Allies then established the Nuremberg Code, being the first international document for voluntary human consent for research.

With human testing today, the testing proceeds onlyif the patient consents to the study. Though this often leads to people with lesser fortune signing up for human trials to earn some extra cash. The ethics of the entire research situation can still be hotly debated.

Military weapon development is another major crossroad of science and ethics. Take, for example, the development of the atomic bombs during the Manhattan Project during WWII. In many ways, the research conducted during these experiments furthered humanity's understanding of atoms, molecules, and quantum. In other ways, this research killed tens of thousands of people.

Military power and weapon technology pose an ethical dilemma largelydue to the nature ofhumankind. If a givencountry doesn't invest resources into developing the best weapons technology, then another more powerful country will simplyswoop in and overpower them. That's the way it works nowadays. It's the unfortunate truth of the interaction of global superpowers. And once again, we're met with a real-life trolley problem.

Do we invest scientific resources into developing better weapons to protect ourselves and thus kill others, or do we let ourselves be killed and "protect" others? We would certainlynot opt for the latter, would we?

Since it seems like the earth has seen better days, maybe it's time to just abandon our planet and move to a new clean slate, like Mars. We know that there is flowing liquid water on Mars somewhere, and we know there are also other resources to help us survive.

So, why not just up and move humanity there?

The biggest ethical questions around Mars colonization are presented when you consider the potential of life on Mars or the potential of future life on Mars. We can't state with absolute certainty that there is life on the planet. Moving humanity there could harm it. We also don't definitively know that life won't occur on the planet through natural means. If humanity moving there interrupts the natural progression of Mars life, isn't that an ethical issue?

RELATED: SPACEX IS PREPARING A MISSION TO COLONIZE MARS BY 2026

The answers to those suppositions largely have to do with how humanity in total should approach its ethical responsibility. If you believe humanity's only ethical responsibility is to themselves, then it's likely not an issue. If you believe that we're responsible for all lesser life forms, then you'll run into countless ethical dilemmasin the process.

Closing out this discussion of ethical dilemmas in science and technology we're left again wonderingwhat are innovation and the betterment of humanity worth? The answer to that question will vary depending upon who you ask... but ask yourself, what is innovation worth?

Originally posted here:
7 Ethically Controversial Research Areas in Science and Technology - Interesting Engineering

Gene editing using the CRISPR system has been established as the most powerful tool in the search for new drugs and is now being exploited for therapeutic purposes. Here, Pushpanathan Muthuirulan discusses the promises and wider opportunities of using CRISPR technology to open up the possibility of large-scale screening of drug targets. He also highlights the importance of implementing CRISPR technology into clinical practice for development of next-generation therapeutics and patient-tailored medicine.

THE DRUG discovery process, in which new drug candidates are discovered and evaluated for therapeutic use, has resulted in both promising and life-saving therapies for numerous diseases including inherited genetic disorders and pathogenic infections.1 However, the discovery and testing of a new drug candidate typically takes more than a decade and the total cost associated with drug discovery processes can exceed $1 billion.2 In the United States, the drug discovery process takes an average of 12 years and in excess of $1 billion to develop a new drug.3 Furthermore, only a few drug candidates actually make it to market; the chance of a new drug actually reaching market is only one in 5,000. The high cost and lengthy effort of getting new drugs to market make the drug development process a risky endeavour for pharmaceutical companies, which consequently hinders discovery and development of new therapies. The recent emergence of genome editing technologies and advances in our understanding of human genome sequences have raised hope that direct manipulation of the genome could potentially revolutionise the process of drug discovery and therapeutics.4 In particular, new technologies like CRISPR-Cas9 are key to unlocking potential drug targets and could have a profound impact on modern drug discovery and development.1,5

Originally posted here:
CRISPR: kick-starting the revolution in drug discovery - Drug Target Review

An artist's illustration of a DNA double helix. New technology makes it easy to edit the human genome, even the germline which can affect all of human evolution. (Image:. U.S. National Human Genome Research Institute/Reuters

By making genetic research so much easier, the recent technology known as CRISPR has allowed scientists an enormous advantage in research into so many areas.

Unfortunately theres a downside which raises serious ethical questions.

Franoise Baylis (CM, ONS, PhD, FRSC, FCAHS), is a research professor at Dalhousie University Halifax who has written on the subject.

The concern with gene-editing is with experiments into modification of human DNA, which could lead in theory to changing of the human genome forever. It could in theory enable creating designer babies. Indeed its been just over a year since the first genome edited babies were created by a Chinese scientist. A Russian researcher has since announced plans to carry out similar human gene editing experiments. Other Chinese experiments involve putting human genes into monkeys. All of these, and other potential experiments, have raised the concern of the scientific world that some of its members are going beyond ethical concerns of science.

Research professor Francoise Baylis at Dalhousie University, Halifax

While some experts have expressed concern about state manipulation to create specific charachteristics, Professor Bayliss suggests that designer babies could result in greater class divide. This would be due to the expense making such technology accessible to the upper echelons and entrenching elite advantage, and thus privilege, in their DNA.

However she says these experiments have resulted in governments and the scientific community coming out with regulations and recommendations to slow down or halt such research until such time as serious ethical discussions take place to establish limits on what should be done and how.

F Baylis on the ethical questions involving CRISPR technology and gene editing of the human genome , Harvard University Press

She says the human genome belongs to all of us and any single or small group of scientists shouldnt be altering it on their own.

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. F Bayliss: Altered Inheritance: CRISPR and the Ethics of Human Genome Editing F Bayliss: Altered Inheritance: CRISPR and the Ethics of Human Genome Editing

Fortunately some action has been taken. The WHO has 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). Professor Bayliss is a member of the group which met for the first time this spring.

She lauds those countries that have established regulations regarding such research but notes there is always the chance of rogue scientists.

She also notes that scientists are usually very concerned about recognition by their peers and being shunned for having crossed established ethical lines is something they would very likely avoid

Additional information

The Conversation: F Bayliss: Dec 10/19: A year after the first CRISPR babies, stricter regulations are now in place

Excerpt from:
CRISPR: Ethics and the gene editing of humans - Radio Canada International (en)

We can already edit genes in human embryos. We can even do it in a way to pass the edits down generations, fundamentally changing a familys genetic makeup.

Doing it well, however, is far more difficult.

Its impossible to talk about human germline genome editing without bringing up the CRISPR baby fiasco. Over a year ago, a rogue Chinese scientist performed an edit on fertilized human embryos that, in theory, makes them resistant to HIV infection. Two twin girls were born, and both had multiple unplanned edits in their genome with unknown health consequencesconsequences that may be passed on to their offspring.

The brash attempt at making scientific history clearly shows that, ethics and morality issues aside, when it comes to germline editingthat is, performing gene edits in egg, sperm, or the embryowere simply technologically not there. Make no mistake: CRISPR may one day wipe out devastating genetic diseases throughout entire family lines, or even the human race. But to harness its power responsibly, there are plenty of technical challenges we need to master first.

This week, Rebecca Lea and Dr. Kathy Niakan at the Human Embryo and Stem Cell Laboratory at the Francis Crick Institute in London, UK, laid out those challenges in a sweeping articlein Nature. CRISPR as a gene editor is getting more specific and efficient by the day, they explained. However, for it to gradually move into germline editing, we also need to understand how the tool tangos with cells during early human development.

The data, they argue, will not only let us zoom into the creation of human life. It will also help inform the debate about potential safe and effective clinical uses of this technology, and truly unlock the doors to the human genome for good.

Correcting dangerous genetic mutations is one reason to pursue germline editing, but CRISPRing human embryos can also unveil insights into the very first stages of human embryo development. Research shows that trying to understand how human embryos form by studying mice might not be the best route, especially when it comes to using those results to tackle infertility and other medical problems. With CRISPR, we have insight into these early stages that were previously completely unattainable. We might only solve infertility issues, but perhaps also allow same-sex couples to have genetic children in the future.

Another argument is that couples already screen for life-threatening mutations during IVF, and using CRISPR on top of that is unnecessary. Not true, the authors argued. When both parents carry a similar mutation that robs them of the ability to have a healthy child, CRISPRnot selection during IVFis the answer. Ultimately, providing more options for patients empowers them to make the choice that is best for their family and circumstances, they said.

This is where it gets complicated.

The big one: were still trying to tease out how CRISPR works in cells that form the embryo, in hopes that we can cut down on potential mistakes.

Let me explain: all cells in the body have a cell cycle, somewhat analogous to a persons life cycle. Many checkpoint life events happen along the way. The cell could decide to divide and have kids, so to speak, or temporarily halt its cycle and stop its own aging. During a cycle, the cells DNA dramatically changes in number and packaging in preparation for its next stage in life.

The problem? The way CRISPR works heavily depends on the cell cycle. Although dubbed an editor, CRISPR actually vandalizes the genome, creating breaks in the DNA strands. What we call gene editing is the cells DNA repair system kicking into high gear, trying to patch up the mess CRISPR left behind. Adult cells that cant be repaired stop their own life cycle at a checkpoint for the greater good. In embryos, however, cells arent nearly as altruistic. Their checkpoints arent fully developed, so they might continue to develop even with severe mutations. Zooming back to the full picture, it means that the resulting early-stage embryo may keep accumulating damage, until it fails in the mothers womb.

To get around this, scientists have tried other ways to push an embryo into accepting a healthy DNA template after a CRISPR snip, which in theory would cut down on unwanted mutations. One idea is injecting the CRISPR machinery at a specific time into fertilized eggs, so it catches the early-stage embryo at just the right time to reduce DNA breaks in both strands. While theoretically possible, the process is kind of like a person trying to jump from a high-speed train into a specific cabin on a rapidly rotating Ferris wheel while blindfolded.

But science is making progress. Although we dont have a detailed movie of cell cycles in human embryos yet, multiple labs are beginning to piece one together, with hopes itll eventually help take off the blindfold when injecting CRISPR. Others are looking into adding CRISPR to sperm before fertilization as an alternative.

At the same time, scientists are also trying to characterize the entire scope of mutations caused by CRISPR. Its not just adding, swapping, or deleting specific letters in genes. Rather, the range of mutations is more complex, including large swaths of genetic rearrangements, unintended cuts relatively far from targeted spots, and other dramatic DNA lesions following CRISPR action. Its perhaps not surprising that the edits in CRISPR babies didnt work as intended.

Base editors, which swap one genetic letter for another, might be a better approach compared to the classic hack-and-paste, the authors said. So far, however, the tools havent yet been validated in embryosnot even those from mice.

Finally, for the edit to make a difference to the child, the embryo has to develop normally inside a womb into a baby. But success rates for assisted reproductive technologies are already fairly low. Add in a dose of genetic editing tool that cuts into an already-sensitive genomic landscape, and it becomes incredibly hard to maintain the health of the edited embryo.

Putting it all together, there is simply not enough data at present to understand the capability of early[embryos] to repair DNA, the authors said.

Far from it. Although theres much we dont yet understand, we do have an impressive range of tools to predict and evaluate mutations in human embryos. Exactly how to determine whether a gene-edited embryo is healthy remains up for debatefor example, is five unexpected mutations considered ok? What about 500 or 5,000?

That said, just having tools to diagnose the genetic health of an embryo from a tiny bit of DNA is already extremely useful, especially if we as a society decide to move into germline editing as a treatment.

With machine learning making an ever-larger splash in computational biology, these predictive tools will only become more accurate. Add to that ever-more-effective CRISPR variations, and were on the right trackas long as any potential applications of embryo editing only come after in-depth public and policy discussions and fit a number of strict ethical and safety criteria, the authors said.

In response to the CRISPR baby scandal, multiple governments and the World Health Organization have all drafted new guidelines or legislation to tap on the brakes. The technology isnt mature enough for clinical use, the authors said, and much more work is needednot just to further improve CRISPR tools, but especially for understanding how it works in human embryos.

Ultimately, were talking about potentially engineering the future of the human race. Tiptoeing, rather than stumbling ahead, is the least we can do. One must ensure that the outcome will be the birth of healthy, disease-free children, without any potential long-term complications, the authors concluded.

Image Credit: Image by marian anbu juwan from Pixabay

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How Far Are We from (Accurately and Safely) Editing Human Embryos? - Singularity Hub

Increase in applications of CRISPR and Cas gene editing technology in bacteria and usage of gene editing technology for prevention of various diseases are the major factors anticipated to drive the market from 2018 to 2026. Rise in need of alternative medicine for chronic diseases and increase in investments by key players in Asia Pacific are projected to propel the market during the forecast period.

The report also provides profiles of leading players operating in the global CRISPR and Cas market such as Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc.

To garner compelling insights on the forecast analysis of CRISPR and Cas Genes Market, Request a PDF Sample Here https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=26417

Increase in Usage of DNA-free Cas

DNA-free Cas9 is most commonly used with synthetic crRNA tracrRNA and chosen by researchers who strive to avoid unwanted vector DNA integration into their genomic DNA. CRISPR-Cas9 utilizing mRNA or protein is ideal for applications such as knocking of a fluorescent reporter using HDR or knockout cell line generation. Advantages such as gene editing with DNA-free CRISPR-Cas9 components to reduce potential off-targets and potential usage of CRISPR-Cas9 gene editing to find correlations with human diseases in model systems drive the segment.

Rise in Incidence of Genetic Disorders and Increase in Applications of CRISPR and Cas Genes to Propel Market

Genetic diseases are generally termed as rare diseases. According to NCBI, prevalence of these rare diseases is approximately 5 in 10,000. There are 6,000 to 8,000 rare diseases, with 250 to 280 new diseases diagnosed every year. Hence, 6% to 8% of the global population is projected to be affected by rare diseases i.e., genetic diseases in the near future. Researchers are developing treatments for these diseases with applications of new technologies such as CRISPR. The applications of CRISPR technology are expanding in other industrial sectors. This is expected to drive the market during the forecast period.

Usage of CRISPR/Cas9 technology in plant research has enabled the investigation of plant biology in detail which has helped to create innovative applications in crop breeding. Site-directed mutagenesis and site-specific integration of a gene, which is also called knock-in, are important in precision crop breeding. Cas9/gRNA-mediated site-directed mutagenesis and knock-in is widely used in rice and Arabidopsis protoplasts. CRISPR/Cas9 provides a simple method to generate a DSB at a target site to trigger HDR repair.

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Asia Pacific Market to Witness Exponential Growth

In terms of revenue, the CRISPR and Cas genes market in Asia Pacific is expected to expand at a CAGR of 22.0% during the forecast period. Growth of the market in the region can be attributed to increase in incidence of chronic diseases such as cancer and the need of development of genetic engineered treatment options. According to the report, Call for Action: Expanding Cancer Care for Women in India, 2017, an estimated 0.7 million women in India are suffering from cancer. China dominated the CRISPR and Cas genes market in Asia Pacific. In 2016, scientists based in China launched the first known human trials of CRISPR, the genomic tech that involves slicing and dicing the bodys very source code to fight cancer. Japan was the second largest market for CRISPR and Cas genes in Asia Pacific.

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CRISPR and Cas Genes Market will Likely Rise to US$ 7,234.5 Mn by the End of 2026 - Montana Ledger

In the wake of the Conservative Partys crushing victory in the election in the United Kingdom, Prime Minister Boris Johnson is poised to navigate Britains exit from the European Union. Once out of the EU, the UK could regain full control over its laws and regulations. And that might open the door to a reversal on what scientists consider its backward-looking policies on GMOs and CRISPR gene editing in agriculture.

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, Johnson hadpromised 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

View original post here:
Viewpoint: With Conservative sweep of the 'Brexit election', Boris Johnson poised to steer the UK out of 'outdated' EU GMO, CRISPR regulations -...

We are still in an overall bull market and many stocks that smart money investors were piling into surged through the end of November. Among them, Facebook and Microsoft ranked among the top 3 picks and these stocks gained 54% and 51% respectively. Hedge funds' top 3 stock picks returned 41.7% this year and beat the S&P 500 ETFs by 14 percentage points. Investing in index funds guarantees you average returns, not superior returns. We are looking to generate superior returns for our readers. That's why we believe it isn't a waste of time to check out hedge fund sentiment before you invest in a stock likeCRISPR Therapeutics AG (NASDAQ:CRSP).

CRISPR Therapeutics AG (NASDAQ:CRSP) was in 16 hedge funds' portfolios at the end of September. CRSP investors should be aware of an increase in enthusiasm from smart money recently. There were 13 hedge funds in our database with CRSP positions at the end of the previous quarter. Our calculations also showed that CRSP isn't among the 30 most popular stocks among hedge funds (click for Q3 rankings and see the video below for Q2 rankings). Video: Click the image to watch our video about the top 5 most popular hedge fund stocks.

5 Most Popular Stocks Among Hedge Funds

So, why do we pay attention to hedge fund sentiment before making any investment decisions? Our research has shown that hedge funds' small-cap stock picks managed to beat the market by double digits annually between 1999 and 2016, but the margin of outperformance has been declining in recent years. Nevertheless, we were still able to identify in advance a select group of hedge fund holdings that outperformed the Russell 2000 ETFs by 40 percentage points since May 2014 (see the details here). We were also able to identify in advance a select group of hedge fund holdings that underperformed the market by 10 percentage points annually between 2006 and 2017. Interestingly the margin of underperformance of these stocks has been increasing in recent years. Investors who are long the market and short these stocks would have returned more than 27% annually between 2015 and 2017. We have been tracking and sharing the list of these stocks since February 2017 in our quarterly newsletter. Even if you aren't comfortable with shorting stocks, you should at least avoid initiating long positions in stocks that are in our short portfolio.

We leave no stone unturned when looking for the next great investment idea. For example Europe is set to become the world's largest cannabis market, so we check out this European marijuana stock pitch. One of the most bullish analysts in America just put his money where his mouth is. He says, "I'm investing more today than I did back in early 2009." So we check out his pitch. We read hedge fund investor letters and listen to stock pitches at hedge fund conferences. We also rely on the best performing hedge funds' buy/sell signals. We're going to take a glance at the fresh hedge fund action surrounding CRISPR Therapeutics AG (NASDAQ:CRSP).

At Q3's end, a total of 16 of the hedge funds tracked by Insider Monkey were long this stock, a change of 23% from one quarter earlier. On the other hand, there were a total of 16 hedge funds with a bullish position in CRSP a year ago. So, let's examine which hedge funds were among the top holders of the stock and which hedge funds were making big moves.

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Is CRSP A Good Stock To Buy?

More specifically, Cormorant Asset Management was the largest shareholder of CRISPR Therapeutics AG (NASDAQ:CRSP), with a stake worth $44.4 million reported as of the end of September. Trailing Cormorant Asset Management was Farallon Capital, which amassed a stake valued at $23 million. OrbiMed Advisors, Clough Capital Partners, and Valiant Capital were also very fond of the stock, becoming one of the largest hedge fund holders of the company. In terms of the portfolio weights assigned to each position Cormorant Asset Management allocated the biggest weight to CRISPR Therapeutics AG (NASDAQ:CRSP), around 2.71% of its 13F portfolio. Clough Capital Partners is also relatively very bullish on the stock, dishing out 1.63 percent of its 13F equity portfolio to CRSP.

Now, key hedge funds were leading the bulls' herd. OrbiMed Advisors, managed by Samuel Isaly, assembled the largest position in CRISPR Therapeutics AG (NASDAQ:CRSP). OrbiMed Advisors had $21.2 million invested in the company at the end of the quarter. Steven Boyd's Armistice Capital also made a $10.8 million investment in the stock during the quarter. The other funds with new positions in the stock are William Harnisch's Peconic Partners, Israel Englander's Millennium Management, and Sculptor Capital.

Let's check out hedge fund activity in other stocks similar to CRISPR Therapeutics AG (NASDAQ:CRSP). We will take a look at Fastly, Inc. (NYSE:FSLY), The Hain Celestial Group, Inc. (NASDAQ:HAIN), TeleTech Holdings, Inc. (NASDAQ:TTEC), and Chart Industries, Inc. (NASDAQ:GTLS). This group of stocks' market caps match CRSP's market cap.

[table] Ticker, No of HFs with positions, Total Value of HF Positions (x1000), Change in HF Position FSLY,16,126686,3 HAIN,19,545805,2 TTEC,14,41238,-3 GTLS,19,261236,-5 Average,17,243741,-0.75 [/table]

View table hereif you experience formatting issues.

As you can see these stocks had an average of 17 hedge funds with bullish positions and the average amount invested in these stocks was $244 million. That figure was $150 million in CRSP's case. The Hain Celestial Group, Inc. (NASDAQ:HAIN) is the most popular stock in this table. On the other hand TeleTech Holdings, Inc. (NASDAQ:TTEC) is the least popular one with only 14 bullish hedge fund positions. CRISPR Therapeutics AG (NASDAQ:CRSP) is not the least popular stock in this group but hedge fund interest is still below average. Our calculations showed that top 20 most popular stocks among hedge funds returned 37.4% in 2019 through the end of November and outperformed the S&P 500 ETF (SPY) by 9.9 percentage points. A small number of hedge funds were also right about betting on CRSP as the stock returned 74.8% during the first two months of Q4 and outperformed the market by an even larger margin.

Disclosure: None. This article was originally published at Insider Monkey.

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