Archive for the ‘Crispr’ Category

In her three decades in science, Jennifer Doudna said she has seen a gradual erosion of trust in the profession, but the recent Nobel Prize winner told "Axios on HBO" that the institution itself has been under assault from the current administration.

Why it matters: That has manifested itself in everything from how the federal government approaches climate change to the pandemic.

The big picture: Doudna acknowledges that the scientific community probably hurt itself. The effort to stay above the political fray may well have led to too little dialogue between those making discoveries and the leaders responsible for funding those efforts.

Driving the news: Doudna and French colleague Emmanuelle Charpentier were earlier this month awarded the Nobel Prize in chemistry for their work on CRISPR, a gene-editing technique that can be likened to a pair of molecular scissors that can change DNA.

The bottom line: While Doudna was part of the first all-female team to win the Nobel Prize in chemistry, she said her goal is for that to eventually be unremarkable.

Originally posted here:
CRISPR pioneer: "Science is on the ballot" in 2020 - Axios

Scientists Emmanuelle Charpentier and Jennifer Doudna have won the Nobel Prize in chemistry for their pioneering work on the gene-editing tool CRISPR. The tool has been used to engineer better crops and to try to cure human diseases. (Oct. 7) AP Domestic

Coronavirus tests performed in labsare the gold standard for accuracy, and antigen tests are a fast and inexpensive alternative.

But backers of a third type of test, developed by a Nobel Prize winner usingcutting-edge CRISPR technology, say it has the potential to be all three:rapid, accurate and inexpensive.

Although these gene-editing technology testsare still being developed and won't be readyin the United States this yearas the weather cools and demandsurges, research groups recently published papers describing them as anappealing alternative as testing shortages persist amid the COVID-19 pandemic.

Dr. Jennifer Doudna, a University of California-Berkeley researcher whose pioneering work in CRISPR earned a share of this year's Nobel Prize in chemistry, said the test can be done quickly and doesn't require a lab.

"We have a ways to go before CRISPR-based diagnostics reach widespread use, but I believe well see an impact during the current pandemic," Doudna said. "Because it is simple to adjust these tests to detect other targets, the platform were developing now is laying the groundwork to deploy CRISPR for rapid diagnosis during future outbreaks."

Dr. Jennifer Doudna of the University of California-Berkeley earned a share of this year's Nobel Prize in chemistry for her pioneering work in CRISPR technology.(Photo: Susan Walsh, AP)

CRISPR, or clustered regularly interspaced short palindromic repeats, is a gene-editing technology studiedfor a wide range of uses from cancer and sickle cell disease treatments to improved food production.

In 2016, Doudna's lab developed a way to detect RNA using the technology.Her lab collaborated with Dr. Melanie Ott of San Francisco-based Gladstone Institutes to develop an HIV test, but when the pandemic hit, the researchers focused on developing a coronavirus test.

The test recognizesa sequence of RNA inSARS-CoV-2, the coronavirus that causes COVID-19.

Inan Oct. 12 publication, researchers reported the test yielded results infive minutes and correctly identified five samples from patients with the coronavirus. When used witha mobile phone to detect signals generated by thetest, the technology couldprovide a fast, low-cost test outside a laboratory, researchers saidin the paper, which was not peer-reviewed.

Massachusetts Institute of Technology scientists also are honing a CRISPR-based test that can be used outside a lab. In a New England Journal of Medicine letter published last month, researchers said the test was evaluated at a University of Washington lab using 202 samples with the coronavirus and 200 without. The test correctly identified 93.1% of positive samples. The test also had 98.5% specificity, which means it rarely reportedfalse positives.

Feluda, a paper-based CRISPRtest named after a fictional India detective, has been cleared by that nation'sdrug regulators for commercial launch. But it's unclear how the Indian conglomerate Tata Group plans to deploy the test in India, which trails only the United States with nearly 7.7 million cases, according to data from Johns Hopkins University.

GigiGronvall, a senior scholar at the Johns Hopkins Center for Health Security, said she expects more labs will explore tests using this technology.She called it"extremely promising" because people can use thetests outside the lab.

"That has been a big challenge with testing generally,"Gronvall said."People need to have their results pretty quickly otherwise they keep going about their day and they might be infectious and not stop having contact with other people."

More: White House COVID-19 outbreak shows the limits of testing; even the most accurate ones can miss the coronavirus early

More: Rapid, cheap home tests: Companies attempt to make coronavirus tests widely available

More: 'Totally unacceptable': Testing delays force labs to prioritize COVID-19 tests for some, not others

The south San Francisco-based biotechnology firm Mammoth Biosciences, co-founded by Doudna, is working to further develop its test and make it available to labs and medical providers. The companyreceived a National Institutes of Health grant to accelerate development.

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Mammoth CEO Trevor Martin said the company's goal is to make a test that is fast and accurate.

"Right now you have to make this choice: Do you want something that is simple and fast or something that has the highest accuracy?" Martin said.

The most accurate lab tests are molecular-basedpolymerase chain reaction, or PCR, which amplify a small amount of genetic material from anasal swab sample. Labs are limited in the number of PCR tests they can run because of periodic shortages of chemical reagents and other testing materials. In July, when demand outpaced labs' ability to perform tests, consumers whose tests were routed to major labs routinely waited a week or longer for results.

Antigen tests, which detect proteins of the coronavirus,can produce results in 15 minutes but are considered less sensitive than lab tests.

Martin saidCRISPR-based tests offer "a technology that is very simple, very fast yet extremely accurate."

Beyond the technology, test developers are trying to solve another challenge: designinga test that is easy to use and inexpensive.

Dr. Feng Zhang, an MIT biochemist and core member of the Broad Institutewhose lab is developing the StopCOVID test, said his team wants to make a low-cost device that can work with a disposable cartridge. Collaborators wanta testeasy enough for consumers to use at home by takingtheir own nasal swab.

"The challenge is to work out how to scale up the manufacturing so that we can get the cost to be as low as possible," Zhang said.

He said his collaborators are working to bring the test to market as soon as possible. A version of the test already is being used by a Thailand hospital to screen patients.

Mammoth's Martin said designing a test for wide useis "where a lot of things stumble."

"Its really nice to have a new technology but you need to get it to the places where its needed," he said. "Thats something weve been working on very diligently."

Dr. Sophia L. Yohe,director of the University of Minnesotas Molecular Diagnostics Laboratory, said it'sunknown whether test developers will be able to streamline steps to allow a large number of tests.

Some early versions of CRISPR tests required samples to be extracted and amplified, similar to a lab-based PCR tests, saidYohe, chair of the College of American Pathologists Personalized Health Care Committee.

"If you can do only one test at a time, even if it only takes 30 minutes, suddenly doing a thousand tests takes a long time," she said. "So scalability I think is an issue that has to be considered if you are looking at doing high volumes of testing."

Ken Alltucker is on Twitter as@kalltuckeror can be emailed at alltuck@usatoday.com

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Fast, cheap, accurate: Researchers pin hopes on Nobel Prize-winning CRISPR technology to detect coronavirus - USA TODAY

[MUSIC - THEME, SWAY]

(SINGING) When you walk in the room, do you have sway?

Jennifer Doudna just won the Nobel Prize for Chemistry. Its for her work on something called Crispr. Shes the smart one so shell explain it in a minute. Doudna and her collaborator, Emmanuelle Charpentier, are only the sixth and seventh women in history to win chemistrys highest honor. Their work is groundbreaking, a fast and precise way to edit the genome. Its already been used to grow seedless tomatoes, double a dogs muscle mass, and treat people with sickle cell anemia. Someday, it could be used to make designer babies. But before we get to the complex ethics of playing God, we started with the basics.

Lets explain the basic idea of Crispr. It stands for and I want you to say it because youll say it correctly, and Ill bollocks it. But it stands for

Clusters of Regularly Interspaced Short Palindromic Repeats. Say that three times fast. [LAUGHS] Or dont.

You lost me at palindromic. But I know what a palindrome is, so thank goodness. So explain very basically how its different from before. Because you didnt discover Crispr. Francisco Mojica did. But talk about this breakthrough now.

Well, I think its important to point out that in bacteria, Crispr works as an adaptive immune system, analogous to the way our own bodies fight infection. The mechanisms are different, but the principle is the same. Our bodies can adapt to viruses, learn how to fight them. And similarly, in bacteria, the bacteria use the Crispr pathway to do that. The characteristic of all of these Crispr immunity systems is a distinctive pattern of DNA sequences that represents the storage mechanism for learning about a new virus, and then learning how to fight back against it.

So its bacteria protecting itself.

Exactly, yep. And I was interested in this because I thought it was incredibly exciting that this whole kingdom of life that namely bacteria might have adaptive immunity that had never been studied. Previously, nobody knew about it. And so we wanted to understand it. And this is where the technology comes in because in working with Emmanuelle Charpentier and her team

This is your colleague who you won the Nobel Prize with?

Correct, and so we were able to figure out exactly how this Crispr bacterial immune system operates. And it works using a protein that can cut DNA precisely. And importantly and this is the key to the technology the cutting, the position, the sequence of DNA that gets cut is defined by a small molecule of RNA, which is a chemical cousin of DNA that can be, after our work, controlled by scientists to allow this Crispr protein called Cas9 to cut DNA at a place of our choosing.

Right, and youre injecting this RNA in, correct?

Yeah, there are different ways to put it in. You can inject it. You can program the cell to make it. Its rewriting the code. Its taking an editor to the code, like you might edit a Word document. And so that just gives scientists the ability to address questions they couldnt address before. And I think that its fair to say that human beings now have a tool for manipulating DNA precisely in cells. That puts in our hands the ability to control our fate, control our genetic fate, and that of all other organisms that we occupy the planet with. So it is a profound opportunity and also a big challenge to make sure that its used responsibly.

And so essentially, when you were discovering this, when this was your breakthrough, you compared it at one point to a good suspense novel. Can you explain that?

Well, science for me is always a good suspense novel. And this was a particularly interesting volume of it. First of all, we thought, OK, theres evidence that this protein cuts DNA. But how does it do that? And so we did experiments to answer that question. It was a very cool answer. It was, well, this is a programmable protein. We can program it with these RNA molecules to cut a desired DNA sequence. And then I think the leap to the technology was appreciating that we could actually engineer it to be simpler than nature has done by creating a simpler way to make this little RNA molecule that does the programming.

These bacteria were protecting themselves, and then they had a map for you of how they did it.

Yeah, Id call it a map, sure. Yeah, yeah. But heres something very important to appreciate. In bacteria, bacteria do not use Crispr for genome editing, as far as we know. They use it to destroy viral DNA. Thats what they did.

Coming at them.

Its coming at them, and they cut it up and destroy it. But once we understood how that worked, we realized that we could use it differently in plant and animal cells and human cells because of fundamental biological differences in the way those cells deal with DNA breaks.

So essentially, what you invented is gene editing technology, but with scary precision.

Well, I didnt at the time, I wasnt thinking scary. I was thinking, cool, you know? Exciting, wow.

Well get to scary in a minute. But what was the question you asked yourself when you saw that?

First question was, what is it doing? And then once we knew what it was doing, you could start to make connections and say, well, if it does this, then if we put it into these other types of cells, its going to do that. Its going to do genome editing.

So where was the suspense moment?

I think for us, the suspense was kind of twofold. One was, how does it work, and figuring that out. And the second was, is it actually going to be useful for genome editing? And testing that and showing that was just incredibly exciting.

And so you worked with you mentioned is it Dr. Charpentier?

Yeah.

Talk to me about that collaboration. I know Ive heard about you meeting and everything else. How did you work together on it?

Yeah, so we were in three different countries. Emmanuelle was in Sweden at the time. Her student was working Krzys Chylinski working in Vienna and Martin Jinek and I working in Berkeley. So it was a lot of international coordination there, different time zones, et cetera. And it was a lot of fun. It was a lot of fun because the project took off very quickly. They started to get exciting data right away. And so we were communicating back and forth, initially email and occasional Skype calls. Experiments and ideas and thinking would be going on in one time zone, and then wed go to bed. And Emmanuelle and her team would wake up, and they would take over. And we felt like we were sort of working 24/7 that way.

Right. I want to run through some of the high hopes people have for this technology and get it straight, what is actually possible. I kind of think of it, like, you dont know what people are going to make once you invent, say, the iPhone. You dont know what the killer app, so to speak, is. So lets go through some things. Now, could it solve world hunger? How would it do that?

Yeah, no question. I think there is a lot of excitement about that. How do we increase food production? How do we increase the nutritional value of food? I mean, I would just start by saying that when we talk about plants, everything we eat is genome editing, in my opinion, because its been bred to have properties that are valuable to us. And how does that happen? Well, its because plant breeders are introducing random changes into DNA of plants and then selecting for desired traits. So thats been going on for a long time, obviously, for eons, probably. And the difference now with Crispr is that now we have a technology that allows precision. So we dont have to wait for random mutations to crop up, along with all sorts of other things that are maybe undesirable. But we actually can go in and precisely alter a gene or a set of genes and nothing else. And so thats very important.

Drought resistant crops?

Yeah, exactly. In the Innovative Genomics Institute that I founded, which is a Berkeley UCSF partnership, we have a very active program on that. And theyre focused on things like increasing the number of pores in plants or decreasing them to control the amount of water flux into leaves. I mean, its a very practical thing that one can now do with genome editing.

So lets move onto something else. Could you help someone regenerate a lost limb or

Thats going to be tough. Even if we could, for something like that, there are going to be many, many genes involved. So thats part of it. And we dont know what they are. So thats a big jump.

So that would be a challenge. Could you revive extinct animals? The woolly mammoth thing has always seemed to interest scientists.

Yeah, that comes up a lot. I think George Church may disagree with me on this, but I think the wooly mammoth is going to be a big challenge. More power to him if he can do it. But I think

This is a scientist I think at Harvard, right?

Yeah, hes talked a lot about this. I think more realistic, though, is to revive species that have gone extinct recently, like the carrier pigeon, for example, is one that gets discussed. Because it has a genome thats not that different from existing animals, other birds, where you could imagine being able to reengineer changes into an existing genome to recreate the original properties of that bird.

What about future babies smarter, healthier, more beautiful, taller, whatever you want?

Yeah, its a very intriguing idea. Its also one of the most fraught topics in Crispr because of the echoes of eugenics, thinking about safety, and I think just the challenges, honestly, of who decides and how do you monitor health of somebody whos had genes edited when they were an embryo. Its a very big challenge. And so, of course, the field has been working on this for years now in terms of thinking about appropriate regulation and guidelines. And its an ongoing topic. And Im sure many listeners are familiar with the fact that there was an announcement about Crispr babies a couple of years ago that really did galvanize, I think, international cooperation to ensure that that sort of inappropriate use doesnt happen again.

Right, so a couple more of the future possibilities. Coronavirus something youre working on. How would Crispr be applied to that?

I think its most useful in the current pandemic certainly as a diagnostic method. So this is another one of those things that came out of just doing fundamental biochemistry and understanding the mechanisms of these Crispr proteins, was discovering that some of them have the ability to detect and kind of report on a DNA or RNA sequence that they encounter. We think about it in the context of pandemic preparedness.

So for testing.

Yeah.

For testing, but what about actually putting a gene into people, fixing the gene so they dont get it, rather than flu shots and things like that?

Yeah, interesting possibility. I think not something that will happen in a timeline that will affect the current pandemic, but I think this is a very interesting idea in the future, is, will we understand enough about, for example, how the immune system deals with a virus like the SARS-Cov-2 virus that causes Covid-19 to be able to program cells to get ready?

To get ready before.

Yeah.

So we wouldnt need vaccines. So its vaccination for whatever comes along, correct?

Yeah.

Right, fascinating. So this is this idea of an adaptive immune system. So possibilities, those are more far away, but the day-to-day applications like my brothers a doctor in San Francisco at CPMC, and he has muscular dystrophy. And he was talking about that. Some of these diseases like sickle cell anemia, muscular dystrophy can you talk about things that are in the immediate?

Yeah, well, sickle cell is maybe the one to mention first.

Its a blood disease, right?

Yeah, because its a blood disease. Its also its been well-known for decades that its caused by a single genetic mutation that affects red blood cells. And so Crispr is, thats the right tool one gene, fix the gene. And in fact, that is roaring ahead. There are multiple clinical trials ongoing currently for sickle cell disease. Weve already seen the announcement about Victoria Gray, a patient who received Crispr therapy for her sickle cell disease and has apparently been cured of her disease, which is just extraordinary. In fact, just a couple of days ago, I heard from her doctor on the East Coast writing to me about just his thoughts about it as a technology after the Nobel announcement. So I think many of us feel very excited about those opportunities.

So single gene problems are things that are less complex.

Exactly, and muscular dystrophy is another great example of that, right? Thats another disease well-known, single gene that causes that disease. And Crispr is a tool that can be harnessed for that purpose as well.

Any other areas?

Well, I guess the other two I would mention, one is cystic fibrosis. I think thats a little further down the line because we dont, today, know an easy way to get the gene editing molecules into lung cells, where they would be necessary for cystic fibrosis. But that is a disease where, again, theres a well-known single gene that causes that disease. And then the other area of biology that I think is likely to be impacted by Crispr in the coming 5 to 10 years, I would say, is neurodegenerative disease in the brain. I mean

This is dementia.

Yeah, Dementia, Parkinsons, familial forms of ALS. I mean, these are all diseases where, again, at least in some cases, we understand the gene, or genes. Sometimes its a few genes that are involved. And Crispr in principle could be used to make corrections.

And breast cancer?

Well, breast cancer is harder. I think any cancers, there, I guess, the way I think about Crispr for a cancer treatment is more in the context of cancer immunotherapy as a way to help the immune system fight the cancer.

Yeah. All right, well, lets talk about the darker side of Crispr, obviously. Its something you think about a lot in your book, A Crack in Creation. You describe waking up in a cold sweat from a nightmare. Youre introduced to Adolf Hitler whos wearing a pig mask OK. He wants to know more about your amazing technology. Do you still have nightmares like this? And what do you take away from that?

Yeah. I mean, I think that dream, for me, came kind of relatively early on in the evolution of the development of the technology and, for me, was kind of a crystallization, I guess, of unease that I had and just things that I was thinking about, kind of nebulous fears that kind of all came together in that dream. And I havent had a dream quite like that since, but maybe thats why it really stood out for me. It just it really was kind of this moment, in a way, of, oh my gosh, this

You woke up.

Yeah.

You had this dream, and you woke up, and then did what?

Yeah, and I was sweating. And I just thought, oh my god. I mean, what have I done?

Yeah.

And realized

Its your little Dr. Frankenstein moment, right?

Kind of, yeah. And I just think that, for me, that was one of the kind of stepping stones to getting comfortable, if I ever did. I dont know if I am comfortable, but getting more comfortable, at least, with realizing that, OK, I need to step out of my lab, and I need to start talking about this publicly. Because this is a technology that has great risk.

Right, so lets talk about that. Because, again, we talked about you are fine with gene editing for alterations that arent passed on, that essentially dies with the patient. Then germline editing, which is explain what that is, and why did you want a moratorium, because you declared that five years ago. And it feels a little like Oppenheimer opposing the H bomb.

Well, lets start with what germline editing is. So it means making genetic changes that are heritable. That means they can be passed on to future generations. And so if you introduce a genetic change using Crispr, lets say, in sperm or eggs or an embryo, that is then used for either fertilization or for then implantation and to create a pregnancy, then those genetic changes become part of all the cells in that individual. And they can be passed on to future generations. So its a profound thing to think about because its one thing if youre tweaking a gene for I dont know eye color or something. It sort of sounds pretty innocuous. But its very different if you start thinking about changes that might affect somebodys fertility or their intelligence or their other properties that they might have, even their physical properties. And then how that might be misused, where you can certainly imagine lots of misuses of that.

Oh, one can always. Theres been hundreds of science fiction movies where thats misused, right? Its been imagined.

Its been imagined, yes.

So have you become more flexible on germline? When you just said eye color is innocuous, is it?

Well, is it? Yeah, its a great question. Personally, would I ever use Crispr to change color in a child of mine or advise someone to do that? No. I wouldnt. And I think that with any technology, one has to balance risk versus benefit. And with something like embryo editing, theres still a lot of risk that goes along with it, that inadvertent changes are made. Or even if it was perfect at targeting the gene youre targeting, we dont really know what the long-term effects of a lot of mutations of that nature would actually be in an individual. So I just dont I dont think one could condone that. But I have to say that when I first started thinking about that use of Crispr, I felt really opposed to it. I just thought I just cant I cant see anyone justifying that. But in the intervening years, I guess I have come to appreciate a couple of things. One is that theres a lot of fundamental biology that is not known about early human development that might only be possible to discover using Crispr in embryos that are being utilized for research under appropriate guidelines, and not being allowed to develop beyond a few days, essentially, in the laboratory. And so Ive come to feel that there is value in those kinds of experiments, if theyre conducted under appropriate ethical guidelines. But I certainly dont think that the timing is right or that theres really any justification right now for using Crispr to edit embryos that are then implanted to create a pregnancy.

Right, so this idea of designer humans or genome-engineered humans. What do you think we lose if we start playing with genetics? Do we need genetic variation in life like this? Theyre not imperfections, if you think of it that way.

Absolutely. I mean, I think thats what makes human life so rich, right, is there is all this incredible variation. But I do think that, again, thinking some period of time in the future, lets say that we get to a point where Crispr use in embryos is well understood. Its possible to and we understand genetics of certain diseases. Ill give you one example. There is a gene that is clearly involved in high cholesterol in individuals. And heart disease is still a major killer in many countries, including the U.S. Imagine that you could use Crispr in families that have this gene that makes them susceptible to heart disease to give them the form of the gene that is protective against that. Would we do it? I think its possible, right? I think when the technology is clearly safe and were making that one tweak to one gene, I think that could be something that some families might decide they want to do.

Who decides, from your perspective, when its dangerous? Because the National Security Agency released a report on threat assessment and include genome editing in there as a potential weapon of mass destruction.

Yeah.

What scares you the most about where the technology could lead, without any laws in place?

When I think about how it could get rolled out, I think it could happen in the context of in vitro fertilization clinics that, for example, offer a menu of traits and say, check off. I want my baby to have this, that, the other thing. And then were going to use Crispr to make those tweaks. I think that makes me uncomfortable certainly now, partly because, technologically, its just not realistic to do that without a lot of risk, but also because of what you just said. Who should decide that? And how would you monitor the health of individuals who were born after that kind of treatment? And I think these are very big questions

So who should?

that have yet to be addressed.

Who should, from your perspective?

I guess I do feel that if we look at the history of in vitro fertilization, that does offer a road map. Is it the right one? Im not sure, but it is a roadmap. And as you may know, that technology developed, in many ways, in kind of a very grassroots sort of way. There was no top down prescription of, heres how were going to do it. It was more just initial efforts to use it, kind of in a one-off way. Louise Brown, of course, a very famous first baby born as a result of IVF. And when it was clear that she had sort of developed normally, I think many people that were facing infertile lack of fertility, this became a very realistic option. So I think that we could see Crispr getting deployed in a similar fashion, where there will be kind of one-off uses here and there. And depending on the outcomes, that will start to be more widely adopted. [MUSIC PLAYING]

Well be right back.

Two years ago, a Chinese scientist shocked the world. A pair of twins had been born to an HIV positive father. And Dr. He Jiankui announced he had modified their embryos to make the newborns HIV resistant. They were the worlds first Crispr babies. The scientist who engineered them was found guilty of illegal medical practices and is currently serving a three-year prison sentence in China.

What levers and regulations exist in the scientific community?

Well, lets start with here in the U.S. In the United States, since the 1970s, weve had regulations in place that really make it impossible for anyone here to get regulatory approval to create a pregnancy. So what I understood from talking to the scientist who reported this, He Jiankui, is that I think he really felt that this would be OK. I think that he didnt think that he was breaking any laws or that he was doing anything illegal at the time. I think he saw himself, in a way, as somebody who was bringing this technology to people that might, in the future, want to use it with their babies.

Yeah, and he e-mailed you to tell you about it. Is that correct?

He did, yeah.

Can you recap that? Youre getting that email. What did you think?

Yeah, well, I got that email. I was actually here in the U.S. I was about to leave for a conference on human genome editing, the second international summit, which was happening in Hong Kong. This was two Novembers ago. And it was Thanksgiving weekend, in fact. And I received an email with a subject line, Babies Born.

Oh, no.

And yeah, it was kind of one of those nightmare emails that came in.

But not a nightmare, a real one.

View original post here:
Kara Swisher and Dr. Jennifer Doudna on CRISPR and Its Possibilities - The New York Times

It all began with bacteria: The observation and study of these single-celled organisms in the early 1980s allowed scientists to get a glimpse of the bacterial genome. In bacterial DNA, researchers found a class of repetitive nucleotide sequences, which they called Clusters of Regularly Interspaced Short Palindromic Repeats, or CRISPR.

This finding was the basis of what would become CRISPR-Cas9, the gene editing tool that won this years Nobel Prize in Chemistry, awarded to Dr. Emmanuelle Charpentier and Dr. Jennifer Doudna, who began their work in 2011.

Like human cells, bacteria have the ability to recognize invading pathogens that attack their DNA. Unlike human cells, which have a variety of ways to kill viruses or inhibit their replication, some bacteria incorporate a part of the virus genome into their own DNA, using it as a guide to detect DNA from similar viruses during subsequent infections. These viral segments are known as spacers and are integrated into the bacterias CRISPR sequences for later use.

The other component of the CRISPR system is Cas9, a bacterial enzyme that cleaves foreign DNA marked by CRISPR sequences. If the bacterial cell detects the same virus a second time, a copy of the viral DNA is transcribed to the Cas9 enzyme, giving it a molecular clue of what to search for in the cell. Once it matches the clue sequence to the invaders DNA, Cas9 acts like a pair of scissors, cutting the viral DNA and preventing reinfection.

While working with onebacterial species, Charpentier found RNA sequences which play a key role in the cleavage mechanism of CRISPR-Cas9. In collaboration with Doudna, they succeeded in recreating the Cas9s genetic scissors in a test tube, showing that upon fusing an RNA transcript (also called a guide RNA) with Cas9, they could develop a simplified system that reproduces that same cleavage mechanism.

Over the next 10 years, Charpentier and Douda refined their method of gene editing, allowing them to excise and insert DNA sequences of their choosing. In an interview with The McGill Tribune, Dr. Yann Joly, a bioethicist and assistant professor in McGills Department of Human Genetics, commented that their discovery would revolutionize the idea of gene editing.

Charpentier and Doudna used this knowledge about an obscure bacterial immune mechanism (CRISPR) and transposed it into a tool that can simply and cheaply edit the genomes of everything living organism: Human, animal, or vegetable, Joly said.

The advantage of this method is that it enables not only cutting, but also the pasting of new DNA sequences at the site of the excision. Guide RNAs can also provide a template for repairing broken DNA.

Many genome editors existed before CRISPR, but using them was time consuming, cumbersome, and expensive, Joly said.

A technology that has the power to edit the genetic makeup of humans raises ethical concerns.

Because CRISPR-Cas9 is so easy to access and use by anyone with [any] scientific knowledge, it means that the technology is also vulnerable to misuse by biohackers and bioterrorists, Joly said. Given potential limitations and [possible] side-effects of the technology, along with the potential for editing the germline, the consequences could be far reaching. Such misuses are also very difficult to prevent through traditional ethic[s] policies and regulations.

One current debate discusses the extent to which CRISPR-Cas9 should be regulated in clinical research involving human subjects. Although there are legal implications, this discovery has potential for revolutionizing the field of gene therapy and targeted medicine. Regardless, CRISPR-Cas9 still has a long way to go, both mechanically and ethically, before its use can become widespread.

The potential [uses of CRISPR-Cas9] in medicine and agriculture [are] extremely promising but the technology still needs a lot of fine tuning, Joly said. Research and experiments on computer models and animal models [remain important to understanding] off-target effects, [such as] mosaicism and unknown long-term effects.

Originally posted here:
CRISPR-Cas9: The gene editing tool that has revolutionized molecular biology - McGill Tribune

Babies born with a faulty maternal copy of the UBE3A gene will develop Angelman syndrome, which currently has no cure, and has only limited treatments. Now, scientists have shown that gene editing and gene therapy techniques can be used to restore the UBE3A in human neuron cultures and treat deficits in an animal model of Angelman syndrome.

The study, led by senior author Mark Zylka, PhD, Director of the UNC Neuroscience Center and W.R. Kenan, Jr. Distinguished Professor of Cell Biology and Physiology, has been published in the journal Nature, and lays important groundwork for a long-lasting treatment or cure for this debilitating disease, as well as a therapeutic path forward for other single-gene disorders.

Angelman syndrome is caused by a deletion or mutation of the maternal copy of the gene that encodes the ubiquitin protein ligase E3A (UBE3A). The paternal copy of UBE3A is typically silenced in neurons, so the loss of maternal UBE3A results in a complete absence of the UBE3A enzyme in most areas of the brain. This enzyme targets proteins for degradation, a vital process that maintains normal function of brain cells.

When that process malfunctions, the result is Angelman syndrome, a brain disorder with symptoms that include severe intellectual and developmental disabilities, seizures, and problems with speech, balance, movement, and sleep.

Our study shows how multiple symptoms associated with Angelman syndrome could be treated with a CRISPR-Cas9 gene therapy, Zylka said. And we are now pursuing this with help of clinicians at UNC-Chapel Hill. Turning on the paternal copy of UBE3A is an attractive therapeutic strategy because it could reverse the underlying molecular deficiency of the disease.

However, the paternal gene is silenced by a long strand of RNA, produced in the antisense orientation to UBE3A, which blocks production of the enzyme from the paternal copy of the gene.

Members of the research team set out to devise a way to use CRISPR-Cas9 to restore the UBE3A enzyme to normal levels by disrupting the antisense RNA.

The team used an adeno-associated virus (AAV) gene therapy to deliver the Cas9 protein throughout the brain of embryonic mice that model Angelman syndrome. Because UBE3A is essential for normal brain development, early treatment is crucial.

The researchers found that embryonic and early postnatal treatment rescued physical and behavioural phenotypes found in Angelman syndrome patients, finding that a single neonatal injection of AAV unsilenced paternal UBE3A for at least 17 months. The data suggests this effect is likely to be permanent, and the researchers also demonstrated that this approach was effective in human neurons in culture.

Zylka said: No other treatments currently being pursued for Angelman syndrome last this long, nor do they treat as many symptoms. I am confident others will eventually recognise the advantages of detecting the mutation that causes Angelman syndrome prenatally and treating shortly thereafter.

The researchers are now focussing on refining an approach that will be suitable for use in humans.

Identifying Angelman syndrome

Using brain imaging and behaviour observations, the Zylka lab will now collaborate with researchers at the Carolina Institute for Developmental Disabilities (CIDD) to identify symptoms in babies that have the genetic mutation that causes Angelman syndrome.

Zylka said: The idea is to use genetic tests to identify babies that are likely to develop Angelman syndrome, treat prenatally or around the time of birth, and then use these early symptoms as endpoints to evaluate efficacy in a clinical trial.

Our data and that of other groups clearly indicates that prenatal treatment has the potential to prevent Angelman syndrome from fully developing.

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Is CRISPR gene therapy for rare Angelman Syndrome on the horizon? - Health Europa

This article was originally published here

J Chem Inf Model. 2020 Oct 27. doi: 10.1021/acs.jcim.0c00929. Online ahead of print.

ABSTRACT

CRISPR-Cas12a is a genome-editing system, recently also harnessed for nucleic acid detection, which is promising for the diagnosis of the SARS-CoV-2 coronavirus through the DETECTR technology. Here, a collective ensemble of multimicrosecond molecular dynamics characterizes the key dynamic determinants allowing nucleic acid processing in CRISPR-Cas12a. We show that DNA binding induces a switch in the conformational dynamics of Cas12a, which results in the activation of the peripheral REC2 and Nuc domains to enable cleavage of nucleic acids. The simulations reveal that large-amplitude motions of the Nuc domain could favor the conformational activation of the system toward DNA cleavages. In this process, the REC lobe plays a critical role. Accordingly, the joint dynamics of REC and Nuc shows the tendency to prime the conformational transition of the DNA target strand toward the catalytic site. Most notably, the highly coupled dynamics of the REC2 region and Nuc domain suggests that REC2 could act as a regulator of the Nuc function, similar to what was observed previously for the HNH domain in the CRISPR-associated nuclease Cas9. These mutual domain dynamics could be critical for the nonspecific binding of DNA and thereby for the underlying mechanistic functioning of the DETECTR technology. Considering that REC is a key determinant in the systems specificity, our findings provide a rational basis for future biophysical studies aimed at characterizing its function in CRISPR-Cas12a. Overall, our outcomes advance our mechanistic understanding of CRISPR-Cas12a and provide grounds for novel engineering efforts to improve genome editing and viral detection.

PMID:33107304 | DOI:10.1021/acs.jcim.0c00929

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Molecular Dynamics Reveals a DNA-Induced Dynamic Switch Triggering Activation of CRISPR-Cas12a - DocWire News

CRISPR Therapeutics (NASDAQ:CRSP), once a high flyer due to its cutting-edge, genome-editing solutions, is one of the more unpopular healthcare stocks this week.

That's almost entirely because of the death of one of the patients participating in an early-stage clinical trial for the company's T-cell cancer-treatment candidate, CTX110, which was reported along with the study's results on Wednesday.

Image source: Getty Images.

Nevertheless, one investment bank continues to be resolutely bullish on CRISPR stock. This is Chardan Capital, whose analyst Geulah Livshits reiterated her buy recommendation in a research note disseminated on Thursday. She believes the unpopular shares will reverse course and has raised their price target to $110 from her previous $100 target -- 22% above the most recent closing price.

According to TipRanks, among 14 analysts tracking the stock, Livshits is one of the more bullish in terms of price target. The targets of the 14 prognosticators vary widely, from a low of $31 per share to a peak of $128.

Interestingly, at the same time the Chardan Capital prognosticator is near the bottom end of estimates for CRISPR's current fourth-quarter net loss. She's estimating that the company will post a shortfall of $1.30 per share. The lowest estimate out of the 17 analysts tracked by Yahoo! Finance is $1.32. On average, the 17 are expecting CRISPR to lose $1.16 per share for the period.

Regarding the CTX110 study, Livshits pointed to the wider results indicating pronounced anti-tumor effects in several of the advanced-stage patients. The analyst wrote in her note that this "could be a positive signal on durability," adding that the candidate has a 42% chance of success.

Nevertheless, CRISPR shares fell on the day, declining by 2.2% against the 0.5% gain of the S&P 500 index.

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Is CRISPR Therapeutics Really Headed to $110? - Motley Fool

Theres a new industry that is rising, that is promising to be as disruptive as AI and blockchain, that industry is genomics. With the benefit of CRISPR, affordable human genome sequencing, and advances in data science, a handful of companies are positioning themselves to take advantage of this new advent of healthcare. We will explore what is genomics, what companies are positioning themselves for this future, and why it matters.

Genomics is the study of a persons genes (the genome), the field reviews the interactions of those genes with each other and with the persons environment. The genome is an organisms complete set of DNA which programs the individual with different physical traits. Virtually every single cell in the body contains a complete copy of the approximately 3 billion DNA base pairs, or letters, that make up the human genome. With the power of data science it is now possible to study how genes interact, and in some cases edit genes in order to modify a single DNA letter, or base, in a gene.

There are multiple exponential technologies that have merged to enable the easy and cost-effective sequencing of the complete human genome. Sequencing the first human genome cost 2.7 Billion and took 13 years. Due to Moores Law and other exponential technologies the cost has now been reduced to $1000, and is projected to be further reduced to $100.

This means we are entering an era where anyone can have their complete human genome sequenced, in search of errors, or predispositions to certain illnesses or health issues.

Multiple companies are clamoring for a marketplace where initially consumers will demand to have their genome sequenced, and eventually, medical professionals will demand it. This is driving the force behind many of the genomic companies in the marketplace, the second driving force is the transformative power of CRISPR technology.

To familiarize oneself with CRISPR, one must first understand that there are multiple use cases for the terminology. In biology, CRISPR refers to DNA sequences naturally found in the genomes of bacteria and other microorganisms. CRISPRs are the sequences of a crucial component of the immune system that protect bacteria from viruses. The CRISPR immune system defends itself from viral attack by destroying the genome of the invading virus. This is where CRISPR technology then enters the scene.

For popular usage and in how we will explain it, CRISPR is shorthand for CRISPR-Cas9. CRISPR in combination with a protein called Cas9 (or CRISPR-associated) is an enzyme that acts like a pair of molecular scissors, capable of cutting strands of DNA. While the idea of gene editing has been around since the 1950s, CRISPR has enabled a cost-effective level of gene editing with a precision that has never before been available.

There are currently dozens of companies that are taking advantage of genomics, we will review five of the leading companies that are publicly traded.

What sets CRSPR Therapeutics apart is the all-star team of founders, this includes Dr. Emmanuelle Charpentier whose seminal research unveiled the key mechanisms of the CRISPR-Cas9 technology, laying the foundation for the use of CRISPR-Cas9 as a versatile and precise gene-editing tool. Numerous awards have recognized her work, including the Breakthrough Prize in Life Science.

CRISPR Therapeutics is developing an efficient and versatile CRISPR/Cas9 gene-editing platform into therapies to treat hemoglobinopathies, cancer, diabetes, and other diseases.

The first therapy that they are advancing is targeting the blood diseases -thalassemia and sickle cell disease, this has now entered clinical testing, as has the first allogeneic CAR-T program targeting B-cell malignancies. While sickle cell is a disease with an arguable small market, once the technology is mature they can advance to targeting other disease vectors.

Invitae Corporation is primed to take advantage of the rapidly decreasing cost of sequencing a human genome. This is a genetic information company that specializes in providing information for genetic diagnostics, preimplantation, and carrier screening for inherited disorders, miscarriage analysis, and hereditary cancer. Basically, any big data that can be provided from sequencing the human genome, Invitae Corporation offers.

Recently, Invitae Corporation partnered with ArcherDX to bring comprehensive cancer genetics and precision oncology to patients worldwide.

For anyone searching to take advantage of a company that is sequencing human genomes, they are a market leader.

Pacific Biosciences of California, Inc. is a biotechnology company founded in 2004 that develops and manufactures systems for gene sequencing and some novel real-time biological observation. Theyve developed a single molecule, real-time (SMRT) Sequencing technology that delivers high consensus accuracy with unprecedented read lengths. This a new way to study the synthesis and regulation of DNA, RNA, and proteins by harnessing advances in biochemistry, optics, nanofabrication, and more.

Some of the current applications they are working on include detecting epigenetic changes during sequencing to open the door to easier exploration of DNA modification to connect genotype with phenotype. This will enable understanding the effects of epigenetics on a broad range of biological processes, including gene expression, host-pathogen interactions, environmental response, DNA damage, and DNA repair. Furthermore, it will uncover the role of epigenetics in the inheritance of traits from one generation to the next.

Twist Biosciences Corp manufactures synthetic DNA for clients in the biotechnology industry. Theyve designed a DNA synthesis platform to address the limitations of throughput, scalability, and cost inherent in legacy DNA synthesis methods. This allows them to achieve precision manufacturing of DNA at scale.

The high-throughput silicon platform enables Twist Biosciences to miniaturize the chemistry necessary for DNA synthesis. This miniaturization allows the company to reduce the reaction volumes by a factor of 1,000,000 while increasing throughput by a factor of 1,000, enabling the synthesis of 9,600 genes on a single silicon chip at full scale.

This is essentially an easy way for other companies to order DNA as needed.

Illumina, Inc develops, manufactures, and markets integrated systems for the analysis of genetic variation and biological function. The company provides a line of products and services that serve the sequencing, genotyping and gene expression, and proteomics markets.

The technology is currently being used to assist with COVID-19, they enable companies working around the clock to track transmission, conduct surveillance, develop therapies and vaccines for any type of viral infection.

Some of their current offerings include access to fast, high-quality, sample-to-data next-generation sequencing (NGS) services such as RNA and whole-genome sequencing services.

While we have listed some of the primary companies that are taking advantage of low-cost human genome sequencing, and gene editing, there are many other companies that are promising. The healthcare industry is ripe for disruption; investors that strategically become shareholders in some of these market leaders will be best positioned for the advent of a new industry. Investors should ensure that they become familiar with genomics, with a special emphasis on CRISPR.

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Investing in Genomics and CRISPR - Everything You Need to Know - Securities.io

CRISPR, the gene-editing device that could someday make deadly diseases a thing of the past, has been the subject of endless fascination and controversy. Now take that and put it on steroids.

Those CRISPR breakthroughs youve heard about (including that scandalous one) were all made possible by the CRISPR-Cas9 system. Cas9 can remove small pieces of harmful DNA with unreal precision, but now an upgrade to the CRISPR-Cas3 systemhas created what could be a miracle or a monster. Cas3 targets DNA, chases it and chomps down an entire strand like Pac-Man eats Power Pellets. Because Cas3 can remove much longer stretches of DNA in pathogenic bacteria quickly and accurately, it could be how we finally figure out to genetically warp pathogens so they no longer make us sick.

Cas3 is a really potent bacterial immune system that targets phagesit can chew up the whole phage genome, UCSF microbiologist and professor Joseph Bondy-Denomy, who recently led a study published in Nature Methods, told SYFY WIRE. Our focus was deleting large non-mobile regions. So we simply started targeting different regions and isolating survivors. We were surprised to see the bacteria survived this and could repair the big cut.

CRISPR actually spawned in bacteria as a defense against their nemeses, viruses known as bacteriophages. Think of bacteriophages as those candy-colored ghosts who want nothing better than to take down Pac-Man. They especially act like Pinky, that pink phantom who is always trying to ambush him in chase mode. Cas3 and Cas9 are both enzymes in the bacterial immune system. When phages threaten bacteria, the bacteria fight back by messing with their genes. They steal some viral DNA and incorporate it into their own, which makes RNA that binds to its DNA mirror image in the phage. CRISPR enzymes will then obliterate the enemy.

Now imagine that a CRISPR enzyme can be manipulated to edit out genetic information in other things besides bacteriophages, including the bacteria that originally evolved this immune system. Having Pac-Man chew up the DNA that makes pathogenic bacteria cause illness could render it harmless.

Cas9 can snap up a small piece of DNA. Cas3 is part of a different bacterial immune system, and behaves more like the pixelated yellow guy with an endless appetite. Think of Pac-Man eating one Power Pellet at a time versus an entire row of them. It chomps on one strand of the DNA double helix and leaves the other strand exposed. When there is an entire strand of harmful DNA that needs to be deleted, what would take Cas9 a hundred bites to erase will take Cas3 just one. This genetic Pac-Man can chow down on as many as 100 bacterial genes. But how exactly does it chew?

Cas3 has a helicase domain which hydrolyzes ATP and pushes it along DNA while it cuts, Bondy-Denomy said.

Meaning, Cas3 is the type of enzyme thatbreaks down ATPadenosine triphosphate, the main source of energy in all life-formsthrough the process of hydrolysis. It reacts with water to break bonds that release high amounts of energy which fuel processes inside cells and makes it possible for those cells to transfer energy from one area to another so an organism can survive. Experimenting with this Pac-Man in a lab and modifying it will enable scientists to study parts of bacterial DNA that have defied understanding. Bacteria often have long strands DNA that originally came from outside sources, and this is where Cas3 comes in. Some of these DNA stretches have no use. Others could be dangerous.

The research team modified the CRISPR-Cas3 system of the pathogenic Pseudomonas aeruginosa and other species of bacteria, some of which were also pathogens. They were able to prove that it was possible for bacteria to function after DNA erasure.

In the future, it could potentially disarm over a thousand types of pathogenic bacteria that swarm in the gut. Not much is known about most of them except food poisoning culprit E.coli and a few others. There are certain genes in these types of bacteria that make you want to hold a paper bag over your face, but imagine if those could be deleted and you never had to worry about food poisoning again. Bondy-Denomy can see even further beyond that.

One of the most promising future uses for Cas3 is deleting large regions of bacterial genomes to screen for new phenotypes, new molecules that get produced, and vaccine production, he said.

Could this potentially be used on the COVID-19 virus? CRISPR systems did originally evolve to fight off viruses, so you never know.

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Pac-Man meets genetics in new DNA editing that plays like the game - SYFY WIRE

The Global CRISPR Technology Market report by Orbis Research provides a complete view of the Global CRISPR Technology Market. The report also comprises market desirability analysis, regional segments, distribution channel, and the route of administration are benchmarked based on market size, market growth, and general attractiveness. The Global CRISPR Technology Market research report offers detailed information which covers market growth, production, consumption, export, revenue, supply, import, market overview, market segmentation and market growth factors analysis. This report contains several drivers and restraints for the Global CRISPR Technology Market with the growth of the market during the forecast period. Likewise, the report includes a comprehensive analysis of segments based on type, application, and geographical regions. The Global CRISPR Technology Market also provides major manufacturers, industry chain analysis, competitive insights, and market share analysis.

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The Global CRISPR Technology Market report by Orbis Research offers a significant view on the market segmentation on the basis of product type, end-user, and regions. These segments have been studied in-depth based on current and future market trends. In terms of geography, the Global CRISPR Technology Market has been sub-segmented into regions such as South America, Europe, Asia, and MEA. The Global CRISPR Technology Market report provides market share analysis along with an in-depth analysis of the major vendors across the globe. Moreover, the report also includes key strategic growth of the global market on the basis of mergers & acquisition, agreements, partnership, distribution channel, collaboration, and regional growth of major vendors in the global as well as local market.

The Major Players Covered in Global CRISPR Technology Market are:The key players covered in this studyThermo Fisher ScientificMerck KGaAGenScriptIntegrated DNA Technologies (IDT)Horizon Discovery GroupAgilent TechnologiesCellectaGeneCopoeiaNew England BiolabsOrigene TechnologiesSynthego CorporationToolgen

Global CRISPR Technology Market by Type:Segment by Type, the product can be split intoEnzymesKitsgRNALibrariesDesign Tools

Global CRISPR Technology Market by Application:Segment by Application, split intoBiomedicalAgricultural

Market segment by Regions/Countries, this report coversUnited StatesEuropeChinaJapanSoutheast AsiaIndiaCentral & South America

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CRISPR Technology Market SWOT Analysis, Latest Trends, By Top Key Players- Thermo Fisher Scientific Merck KGaA GenScript Integrated DNA Technologies...

On Oct. 7, an amazing advancement for women occurred for the first time in history: Emmanuelle Charpentier and Jennifer A. Doudna, a duo of female scientists, received the Nobel Prize in Chemistry without a male contributor listed on the award. They were awarded this prize due to their research in genome editing with the tool called CRISPR. Whether you remember reading about CRISPR in AP Biology in high school or are actively involved with CRISPR as a chemistry or bioengineering student, what remains clear is that these two women paved the way for the next generation of female scientists, and for how genetic diseases will be cured.

The Point talked with a female professor and two female students via email about this advancement. Each had much to share about their experiences as women in this field. Heidi Woelbern, a biology professor at PLNU, uses CRISPR in her research. Olivia Owen and Kaitlyn Morgan are both biochemistry majors.

The Point: What is genome editing and how has Charpentier and Doudnas work changed the course of science in relation to DNA experiments?

Heidi Woelbern: CRISPR technology is revolutionary. The ability to edit genes within the genome is foundational to understanding what exactly they do. When I was in graduate school in the early 2000s, a graduate student might spend two years developing a mouse model in which a single gene was knocked out. For instance, if you were interested in diabetes, you might knock out one of the many genes thought to play a role in diabetes. You would then study the mouse to see if diabetes was ameliorated or then still persisted. With CRISPR technology, a knockout mouse model can be developed in about one month. The gene-editing capabilities are more precise, vastly cheaper and produce results in a fraction of the time.

Olivia Owen: The work of these two women with CRISPR is revolutionary. Its going to be the foundation for so many other scientific and medical breakthroughs, and its already being used in numerous ways to treat cancer and genetic diseases.

Kaitlyn Morgan: I am currently in genetics [class] right now and am learning about CRISPR. It really excited me after learning that these two women were the pioneers of developing the CRISPR model to influence new technological advances in treating disease by altering the genome.

TP: What was your initial reaction when you found out that Charpentier and Doudna had won this prize?

HW: I was thrilled to see this. I love that it was a team of women, working collaboratively, in different countries (Emmanuelle Charpentier was working in Europe, predominantly in Germany and Jennifer Doudna in the U.S.). They shared ideas, their new findings, and even their researchers. It appears (from the outside) to be very collaborative and productive. I love that they both are biochemists. There is so much to learn when one pairs the chemistry and behavior of molecules in isolation and then applies that knowledge into increasingly complex biological models

OO: My initial reaction to finding out that a duo of women won the Nobel prize was excitement. There are so few women in the biochemical engineering field that its so cool to have successful women in the field to look up to.

KM: My initial reaction when finding out that these two women won the Nobel prize in chemistry really motivated me. It reminded me that I am capable and smart enough to pursue a degree in science.

TP: What is your experience in either learning or interacting with CRISPR/genome-editing software?

HW: In collaboration with Dr. Dorrell (biology) and Dr. Jansma (chemistry) we have been working on mutating a gene thought to play a role in cervical cancer. Dr. Jansma has analyzed the E7 oncoprotein from the human papillomavirus. She has been able to determine altered chemical characteristics of E7 based upon some minor changes in the gene (and thus the protein). Using site directed mutagenesis, we are generating these same variants from Dr. Jansmas studies and introducing them into a biologically relevant system to determine if the chemistry can explain the biology. This project thus far has not utilized CRISPR technology. However, we certainly could utilize this technology in the future; especially if we wanted to move into an animal model system.

TP: Are there any female chemists or scientists who are role models for you?

OO: Growing up, I always loved Marie Curie because she was a brilliant chemist even when there were no other women in her field. With these recent advancements in CRISPR, its going to be exciting that young girls are going to have more female role models to look up to.

KM: Honestly, the two women [Jennifer Doudna and Emmanuelle Charpentier] are my role models. The scientific advancement of genome editing that these two women discovered I believe is going to impact the science world by ultimately changing the way doctors treat patients. Overall, these two women have reminded me to keep on working hard and staying focused on my path to pursuing my degree because anything is possible.

Jennifer A. Dounda said in an article from Omniscience, One of the problems in the biotech world is the lack of women in leadership roles, and Id like to see that change by walking the walk.

You can learn more about these women and this years Nobel Prize award ceremony through the official Nobel Prize YouTube channel.

Written By: Elaine Alfaro

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Women in Science: Thoughts on the 2020 Nobel Prize in Chemistry - Loma Beat

A new business intelligence report released by HTF MI with title Global COVID-19 CRISPR Technology Market Insights by Application, Product Type, Competitive Landscape & Regional Forecast 2025 is designed covering micro level of analysis by manufacturers and key business segments. TheGlobal COVID-19 CRISPR Technology Market survey analysisoffers energetic visions to conclude and study market size, market hopes, and competitive surroundings. The research is derived through primary and secondary statistics sources and it comprises both qualitative and quantitative detailing. Some of the key players profiled in the study are Thermo Fisher Scientific (United States), Merck KGaA (Germany), Genscript Biotech (United States), Integrated DNA Technologies, Inc. (United States), Horizon Discovery Group (United Kingdom), Agilent Technologies (United States), Cellecta, Inc. (United States), GeneCopoeia, Inc. (United States), New England Biolabs (United States) and Origene Technologies, Inc. (United States).

Whats keeping Thermo Fisher Scientific (United States), Merck KGaA (Germany), Genscript Biotech (United States), Integrated DNA Technologies, Inc. (United States), Horizon Discovery Group (United Kingdom), Agilent Technologies (United States), Cellecta, Inc. (United States), GeneCopoeia, Inc. (United States), New England Biolabs (United States) and Origene Technologies, Inc. (United States) Ahead in the Market? Benchmark yourself with the strategic moves and findings recently released by HTF MIGet Free Sample Report + All Related Graphs & Charts @:https://www.htfmarketreport.com/sample-report/2833424-global-covid-19-crispr-technology-market

Market Overview of Global COVID-19 CRISPR TechnologyIf you are involved in the Global COVID-19 CRISPR Technology industry or aim to be, then this study will provide you inclusive point of view. Its vital you keep your market knowledge up to date segmented by Applications [Application I, Application II], Product Types [Enzymes, Kits, gRNA, Libraries and Design Tools] and major players. If you have a different set of players/manufacturers according to geography or needs regional or country segmented reports we can provide customization according to your requirement.

This study mainly helps understand which market segments or Region or Country they should focus in coming years to channelize their efforts and investments to maximize growth and profitability. The report presents the market competitive landscape and a consistent in depth analysis of the major vendor/key players in the market along with impact of economic slowdown due to COVID.

Furthermore, the years considered for the study are as follows:Historical year 2014-2019Base year 2019Forecast period** 2020 to 2026 [** unless otherwise stated]

**Moreover, it will also include the opportunities available in micro markets for stakeholders to invest, detailed analysis of competitive landscape and product services of key players.

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The titled segments and sub-section of the market are illuminated below:The Study Explore the Product Types of COVID-19 CRISPR Technology Market: Enzymes, Kits, gRNA, Libraries and Design Tools

Key Applications/end-users of Global COVID-19 CRISPR Technology Market: Application I, Application II

Top Players in the Market are: Thermo Fisher Scientific (United States), Merck KGaA (Germany), Genscript Biotech (United States), Integrated DNA Technologies, Inc. (United States), Horizon Discovery Group (United Kingdom), Agilent Technologies (United States), Cellecta, Inc. (United States), GeneCopoeia, Inc. (United States), New England Biolabs (United States) and Origene Technologies, Inc. (United States).

Region Included are: ** Confirmation on availability of data would be informed prior purchase

Important Features that are under offering & key highlights of the report: Detailed overview of COVID-19 CRISPR Technology market Changing market dynamics of the industry In-depth market segmentation by Type, Application etc Historical, current and projected market size in terms of volume and value Recent industry trends and developments Competitive landscape of COVID-19 CRISPR Technology market Strategies of key players and product offerings Potential and niche segments/regions exhibiting promising growth A neutral perspective towards COVID-19 CRISPR Technology market performance Market players information to sustain and enhance their footprint

Read Detailed Index of full Research Study at @https://www.htfmarketreport.com/reports/2833424-global-covid-19-crispr-technology-market

Major Highlights of TOC:Chapter One: Global COVID-19 CRISPR Technology Market Industry Overview1.1 COVID-19 CRISPR Technology Industry1.1.1 Overview1.1.2 Products of Major Companies1.2 COVID-19 CRISPR Technology Market Segment1.2.1 Industry Chain1.2.2 Consumer Distribution1.3 Price & Cost Overview

Chapter Two: Global COVID-19 CRISPR Technology Market Demand2.1 Segment Overview2.1.1 APPLICATION 12.1.2 APPLICATION 22.1.3 Other2.2 Global COVID-19 CRISPR Technology Market Size by Demand2.3 Global COVID-19 CRISPR Technology Market Forecast by Demand

Chapter Three: Global COVID-19 CRISPR Technology Market by Type3.1 By Type3.1.1 TYPE 13.1.2 TYPE 23.2 COVID-19 CRISPR Technology Market Size by Type3.3 COVID-19 CRISPR Technology Market Forecast by Type

Chapter Four: Major Region of COVID-19 CRISPR Technology Market4.1 Global COVID-19 CRISPR Technology Sales4.2 Global COVID-19 CRISPR Technology Revenue & market share

Chapter Five: Major Companies List

Chapter Six: Conclusion

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Key questions answered What impact does COVID-19 have made on Global COVID-19 CRISPR Technology Market Growth & Sizing? Who are the Leading key players and what are their Key Business plans in the Global COVID-19 CRISPR Technology market? What are the key concerns of the five forces analysis of the Global COVID-19 CRISPR Technology market? What are different prospects and threats faced by the dealers in the Global COVID-19 CRISPR Technology market? What are the strengths and weaknesses of the key vendors?

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CRISPR Technology Market Will Generate New Growth Opportunities in the upcoming year - Aerospace Journal

CRISPR and Cas Genes Market provides an in-depth insight into Sales and Trends Forecast: 2020-2025:

Global CRISPR and Cas Genes 2020 Report consists of important factors such as the latest trends, performance drivers, key players, revenue, growth rate and volume sales, and consumer insights. The most recent report by regal intelligence on global CRISPR and Cas Genes market analyses the impact of COVID 19 on the industry. The reports comprehend the global industry outlook in the light of the current market situation, trends, prominent players in the industry, and how these factors are expected to boost the CRISPR and Cas Genes market during the forecast period. The research study by regal intelligence analyses factors that are dynamic and will affect the CRISPR and Cas Genes market in the near future.

Global CRISPR and Cas Genes market competition by TOP MANUFACTURERS, with production, price, revenue (value) and each manufacturer including

CRISPR Therapeutics, AstraZeneca, Addgene, Caribou Biosciences, Inc., Cellectis, Editas Medicine, Inc., Egenesis, F. Hoffmann-La Roche Ltd., Horizon Discovery Group Plc, Genscrip, Danaher Corporation, Intellia Therapeutics, Inc., Lonza, Merck KGaA, New En

For Better Understanding Go With This Free Sample Report Enabled With Respective Tables and Figures( Get Instant 10% Discount ):https://www.eonmarketresearch.com/sample/72556

Global CRISPR and Cas Genes Market forecast to 2025 providing information such as company profiles, product picture, and specification, capacity, production, price, cost, revenue, and contact information. Upstream raw materials and equipment and downstream demand analysis are also carried out. The Global CRISPR and Cas Genes market trends and marketing channels are analyzed. Finally, the feasibility of new investment projects is assessed and overall research conclusions offered.

Years Considered to Estimate the CRISPR and Cas Genes Market Size:

History Year: 2015-2019

Base Year: 2019

Estimated Year: 2020

Forecast Year: 2020-2025

Region Coverage (Regional Production, Demand & Forecast by Countries, etc.):

CRISPR and Cas Genes Market regional analysis covers the following regions North America, Europe, Asia-Pacific, South America, Middle East & Africa.

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Product Type Coverage CRISPR and Cas Genes Market Size & Forecast, Major Company of Product Type, etc.):

General Type

On the basis of the end users/applications, this report focuses on the status and outlook for major applications/end users, consumption (sales), market share and growth rate for each application, including

Biomedical

Some of the key questions answered in this report:

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Major Point of TOC:

Part I CRISPR and Cas Genes Industry Overview

Chapter One:CRISPR and Cas Genes Industry Overview

Chapter Two:CRISPR and Cas Genes Up and Down Stream Industry Analysis

Part II Asia CRISPR and Cas Genes Industry (The Report Company Including the Below Listed But Not All)

Chapter Three:Asia CRISPR and Cas Genes Market Analysis

Chapter Four:2015-2020 Asia CRISPR and Cas Genes Productions Supply Sales Demand Market Status and Forecast

Chapter Five:Asia CRISPR and Cas Genes Key Manufacturers Analysis

Chapter Six:Asia CRISPR and Cas Genes Industry Development Trend

Part III North American CRISPR and Cas Genes Industry (The Report Company Including the Below Listed But Not All)

Chapter Seven:North American CRISPR and Cas Genes Market Analysis

Chapter Eight:2015-2020 North American CRISPR and Cas Genes Productions Supply Sales Demand Market Status and Forecast

Chapter Nine:North American CRISPR and Cas Genes Key Manufacturers Analysis

Chapter Ten:North American CRISPR and Cas Genes Industry Development Trend

Part IV Europe CRISPR and Cas Genes Industry Analysis (The Report Company Including the Below Listed But Not All)

Chapter Eleven:Europe CRISPR and Cas Genes Market Analysis

Chapter Twelve:2015-2020 Europe CRISPR and Cas Genes Productions Supply Sales Demand Market Status and Forecast

Chapter Thirteen:Europe CRISPR and Cas Genes Key Manufacturers Analysis

Chapter Fourteen:Europe CRISPR and Cas Genes Industry Development Trend

Part V CRISPR and Cas Genes Marketing Channels and Investment Feasibility

Chapter Fifthteen:CRISPR and Cas Genes Marketing Channels Development Proposals Analysis

Chapter Sixteen:Development Environmental Analysis

Chapter Seventeen:CRISPR and Cas Genes New Project Investment Feasibility Analysis

Part VI Global CRISPR and Cas Genes Industry Conclusions

Chapter Eighteen:2015-2020 Global CRISPR and Cas Genes Productions Supply Sales Demand Market Status and Forecast

Chapter Nineteen:Global CRISPR and Cas Genes Industry Development Trend

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CRISPR and Cas Genes Market Insight | Strategic Industry Evolutionary Analysis Focus on Leading Key Players and Revenue Growth Analysis by Forecast To...

CRISPR Technology Marketanalysis is provided for the Global market including development trends by regions, competitive analysis of CRISPR Technologymarket. CRISPR TechnologyIndustryreport focuses on the major drivers and restraints for the key players.

According to the CRISPR Technology Market report, the global market is expected to witness a relatively higher growth rate during the forecast period. The report provides key statistics on the market status of Global and Chinese CRISPR Technology Market manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry

Major Key Contents Covered in CRISPR TechnologyMarket:

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Then, the report explores the international major players in detail. In this part, the report presents the company profile, product specifications, capacity, production value, and 2015-2019 market shares for each company.

After the basic information, the report sheds light on the production. Production plants, their capacities, global production, and revenue are studied. Also, the CRISPR TechnologyMarket Sales growth in various regions and R&D status are also covered.

Through the statistical analysis, the report depicts the global and Chinese total market of CRISPR Technologymarket including capacity, production, production value, cost/profit, supply/demand, and Chinese import/export. The total market is further divided by company, by country, and by application/type for the competitive landscape analysis.

CRISPR TechnologyMarket Report Segmentation:

Product Type:

Application:

Key Players:

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Region Analysis:The report then estimates 2020-2026 market development trends of CRISPR Technologymarket. Analysis of upstream raw materials, downstream demand and current market dynamics is also carried out. In the end, the report makes some important proposals for a new project of CRISPR Technologymarket before evaluating its feasibility.

Table and Figures Covered in This Report:

Then, the report focuses on global major leading CRISPR TechnologyMarket players with information such as company profiles, product picture, and specification, capacity, production, price, cost, revenue, and contact information. Upstream raw materials and downstream consumers analysis is also carried out. Whats more, the Global CRISPR TechnologyMarket development trends and marketing channels are analyzed.

In nutshell, the CRISPR Technology Market feasibility of new investment projects is assessed, and overall research conclusions are offered. In a word, the CRISPR TechnologyMarket report provides major statistics on the state of the industry and is a valuable source of guidance and direction for companies and individuals interested in the Market Sales.

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CRISPR Technology Market 2020: Potential growth, attractive valuation make it is a long-term investment | Know the COVID19 Impact | Top Players:...

The Office of Research and the School of Medicine had planned to introduce their Oct. 30 speaking guest as a professor and the founder and director of the Innovative Genomics Institute at UC Berkeley, and a CRISPR pioneer.

Since being booked for the Distinguished Speaker Series in Research and Innovation, however, Jennifer Doudna has added a new title: Nobel laureate.

She and Emmanuelle Charpentier, director of the Max Planck Institute for Infection Biology, won the Nobel Prize in chemistry Oct. 7 for their co-development of CRISPR-Cas9, a genome editing tool that has revolutionized biomedicine and agriculture.

Whats CRISPR? Jennifer Doudna explains in a Radiolab podcast.

Doudna became the first woman on the UC Berkeley faculty to win a Nobel, and she and Charpentier are the first women to share a Nobel in the sciences.

Doudnas topic for her UC Davis talk is CRISPR and Coronavirus.

UC Davis Healths Ralph Green, distinguished professor in the Department of Pathology and Laboratory Medicine, and medical director of UC Davis Diagnostics, recently collaborated with Doudna and others on a project to set up COVID-19 testing for UC Berkeley and the surrounding community and Green is helping with a similar project at UC Davis.

I had the good fortune to get to know Jennifer Doudna through my interaction with her group when they turned their skills and knowledge to setting up, at remarkable speed, a pop-up, PCR-based test for SARS-CoV-2 during the early days of the COVID-19 pandemic when the country was scrambling to meet the need for more testing, Green said.

I have to say that it has been a singular pleasure and privilege for me to interact with Jennifer Doudna and her colleagues.

CRISPR-Cas9 genetic engineering technology enables scientists to change or remove genes quickly and with great precision. Labs worldwide have redirected their research programs to incorporate this new tool, creating a CRISPR revolution with huge implications across biology and medicine.

Doudna is a leader in public discussion of the ethical implications of genome editing for human biology and societies. She advocates for thoughtful approaches to the development of policies around the use of CRISPR-Cas9.

Follow Dateline UC Davis on Twitter.

See the article here:
'CRISPR and Coronavirus': Hear From Nobel Winner Jennifer Doudna - UC Davis

From the beginning, Pfizer CEO Albert Bourla eschewed government funding for his Covid-19 vaccine work with BioNTech, willing to take all the $2 billion-plus risk of a lightning-fast development campaign in exchange for all the rewards that could fall its way with success. And now that the pharma giant has seized a solid lead in the race to the market, those rewards loom large.

SVB Leerinks Geoff Porges has been running the numbers on Pfizers vaccine, the mRNA BNT162b2 program that the German biotech partnered on. And he sees a $3.5 billion peak in windfall revenue next year alone. Even after the pandemic is brought to heel, though, Porges sees a continuing blockbuster role for this vaccine as people around the world look to guard against a new, thoroughly endemic virus that will pose a permanent threat.

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UPDATED: CRISPR Therapeutics gets a snapshot of off-the-shelf CAR-T success in B-cell malignancies marred by the death of a patient - Endpoints News

Its been a good year for Intellia: One of its founders, Jennifer Doudna, Ph.D., nabbed the Nobel Prize in Chemistry for her CRISPR research.

Now, the biotech she helped build is putting that to work, saying it now plans the worlds first clinical trial for asingle-course therapy that potentially halts and reverses a condition known as hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).

This genetic disorder occurs when a person is born with a specific DNA mutation in the TTR gene, which causes the liver to produce a protein called transthyretin (TTR) in a misfolded form and build up in the body.

How to Streamline Your Clinical Research Organization's Processes End to End

Learn how implementing one platform leads to data consistency and ultimately facilitate faster clinical trials while reducing overall trial costs, leave behind spreadsheets and home-grown tools for a predictable trial and the ability to forecast unit delivery resulting in the optics you need to ensure a successful trial, and hear experts share industry trends of what is affecting the Clinical Research Organization industry today.

hATTR can manifest as polyneuropathy (hATTR-PN), which can lead to nerve damage, or cardiomyopathy (hATTR-CM), which involves heart muscle disease that can lead to heart failure.

This disorder has seen a lot of interest in recent years, with an RNAi approach from Alnylam seeing an approval for Onpattro a few years back, specifically for hATTR in adults with damage to peripheral nerves.

Ionis Pharmaceuticals and its rival RNAi drug Tegsedi also saw an approval in 2018 for a similar indication.

They both battle with Pfizers older med tafamidis, which has been approved in Europe for years in polyneuropathy, and the fight could spread to the U.S. soon.

The drug, now marketed as Vyndaqel and Vyndamax, snatched up an FDA nod last May to treat both hereditary and wild-type ATTR patients with a heart condition called cardiomyopathy.

While coming into an increasingly crowed R&D area, Intellia is looking for a next-gen approach, and has been given the go-ahead by regulators ion the U.K, to start a phase 1 this year.

The idea is for Intellias candidate NTLA-2001, which is also partnered with Regeneron, to go beyond its rivals and be the first curative treatment for ATTR.

By applying the companys in vivo liver knockout technology, NTLA-2001 allows for the possibility of lifelong transthyretin (TTR) protein reduction after a single course of treatment. If this works, this could in essence cure patients of the their disease.

The 38-patient is set to start by years end.

Starting our global NTLA-2001 Phase 1 trial for ATTR patients is a major milestone in Intellias mission to develop medicines to cure severe and life-threatening diseases, said Intellias president and chief John Leonard, M.D.

Our trial is the first step toward demonstrating that our therapeutic approach could have a permanent effect, potentially halting and reversing all forms of ATTR. Once we have established safety and the optimal dose, our goal is to expand this study and rapidly move to pivotal studies, in which we aim to enroll both polyneuropathy and cardiomyopathy patients."

Continue reading here:
Intellia to kick-start first single-course 'curative' CRISPR shot, as it hopes to beat rivals Alnylam, Ionis and Pfizer - FierceBiotech

In a study published [September 21] in theNature Biomedical Engineeringjournal, the researchers said they usedCRISPRCas9 and a combination of other genetic technologies to inactivate porcine endogenous retroviruses (PERVs), a group of viruses that could be dangerous to humans, while also enhancing the pigs immunological and blood-coagulation compatibility with humans, which could reduce the risk of rejection by organ recipients.

The engineered pigs exhibited normal physiology, fertility and transmission of the edited genes to their offspring, according to the paper.

Transplants from pigs have long been investigated as a solution to the global shortage of human organs for patients with organ failure, for reasons such as the size of their organs similar enough to those of humans and their relatively short maturity period of about six months.

The risks of organ rejection due to the biological incompatibility of pig organs with human bodies and of transmitting PERVs have limited the clinical applicability of such transplants, but advancements in gene-editing technology have given researchers new hope.

George Church, one of the authors from Harvard Medical School and a co-founder of Hangzhou-based Qihan Bio, was quoted by Chinese news agency Xinhua as saying that if the technology used can be further verified in future research, it could help alleviate the global shortage of human organs to a large extent.

Read the original post

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CRISPR has brought pig-to-human organ transplants to the cusp of reality - Genetic Literacy Project

The global CRISPR Cas9 Industry Market is carefully researched in the report while largely concentrating on top players and their business tactics, geographical expansion, market segments, competitive landscape, manufacturing, and pricing and cost structures. Each section of the research study is specially prepared to explore key aspects of the global CRISPR Cas9 Industry market. For instance, the market dynamics section digs deep into the drivers, restraints, trends, and opportunities of the global CRISPR Cas9 Industry market. With qualitative and quantitative analysis, we help you with thorough and comprehensive research on the global CRISPR Cas9 Industry market. We have also focused on SWOT, PESTLE, and Porters Five Forces analyses of the global CRISPR Cas9 Industry market.

The research report on CRISPR Cas9 Industry market elaborates on the major trends defining the industry growth with regards to the regional terrain and competitive scenario. The document also lists out the limitations & challenges faced by industry participants alongside information such as growth opportunities. Apart from this, the report contains information regarding the impact of COVID-19 pandemic on the overall market outlook.

Key insights from COVID-19 impact analysis:

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An overview of regional landscape:

Additional information from the CRISPR Cas9 Industry market report:

Table of Contents

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CRISPR Cas9 Industry Market Overview, Environmental Analysis and Forecast to 2025 - Express Journal

A researcher observes the process of genetic scissors in a laboratory in Berlin (Germany). (GREGOR FISCHER / DPA)

The genetic scissors, called Crispr-Case 9 discovered almost ten years ago, are actually genetic code scissors. They make it possible to separate two strands of DNA and replace or delete a gene. When American and French scientists Jennifer Doudna and Emmanuelle Charpentier made this discovery, it paved the way for much research. Today, American, European and Australian scientists are meeting in videoconference to take stock of their progress.

For example, Crispr is making great progress in research into gene therapy. Today, this technique makes it possible to treat people with Duchenne muscular dystrophy, one of the genetic diseases often mentioned during the Telethon. Inserm was also able to reactivate a gene to fight against sickle cell anemia: a blood disease linked again to a genetic problem. Moreover, the company eGenesis works on pigs free of viruses dangerous to humans. The animals would then become perfect organ donors for patients awaiting transplants of the heart, pancreas, etc.

The Crispr technique has developed in many laboratories around the world and two years ago a Chinese researcher, He Jiankui, caused a scandal after announcing that he had used it to create, through in vitro fertilization, two GMO babies resistant to HIV whose father was a carrier. According to a survey, in the MIT Technology Review, the researcher forced the hand of parents who saw in this genetic manipulation the only way to have children without risk. In addition, today it is not known how babies are doing and what other genetic consequences the use of Crispr has had on them. The researcher is still under house arrest in China.

Emmanuelle Charpentier in an interview on point obviously criticizes the failure to respect ethical criteria for the use of its scissors applied to research for humans. On the other hand, she does not share the doubts of peasant and environmental associations on plants modified by Crispr-Cas 9 and considered as GMOs in Europe. For her, her innovation makes it possible to boost a plant gene, to reproduce in an accelerated manner what can happen in nature. Its not like creating mutant plants with a gene that comes from other species.

Applied to theAgriculture, Crispr-Cas 9 makes it possible to create allergen-free peanuts, gluten-free wheat, more drought-resistant rice, but also to remove the horns of cows or to herd only males. Crispr-Cas 9 therefore asks even more to meditate on Rabelais sentence: Science without consciousness is nothing but the ruin of the soul.

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Advances and concerns raised by Crispr-Cas 9, the genetic scissors - Pledge Times

Chemistry Nobel Prize award recipients Jennifer A. Doudna and Emmanuelle Charpentier have joined the ranks of Marie Curie, Frances Arnold, Ada E. Yonath and Dorothy Crowfoot Hodgkin.J.L. Cereijido/EPA

THE Royal Swedish Academy of Sciences yesterday awarded the 2020 Nobel Prize in Chemistry to Emmanuelle Charpentier and Jennifer Doudna for their work on CRISPR, a method of genome editing.

A genome is the full set of genetic instructions that determine how an organism will develop. Using CRISPR, researchers can cut up DNA in an organisms genome and edit its sequence.

CRISPR technology is a powerhouse for basic research and is also changing the world we live in. There are thousands of research papers published every year on its various applications.

These include accelerating research into cancers, mental illness, potential animal to human organ transplants, better food production, eliminating malaria-carrying mosquitoes and saving animals from disease.

Charpentier is the director at the Max Planck Institute for Infection Biology in Berlin, Germany and Doudna is a professor at the University of California, Berkeley. Both played a crucial role in demonstrating how CRISPR could be used to target DNA sequences of interest.

Read more:Why more women dont win science Nobels

CRISPR technology is adapted from a system that is naturally present in bacteria and other unicellular organisms known as archaea.

This natural system gives bacteria a form of acquired immunity. It protects them from foreign genetic elements (such as invading viruses) and lets them remember these in case they reappear.

Like most advances in modern science, the discovery of CRISPR and its emergence as a key genome editing method involved efforts by many researchers, over several decades.

In 1987, Japanese molecular biologist Yoshizumi Ishino and his colleagues were the first to notice, in E. coli bacteria, unusual clusters of repeated DNA sequences interrupted by short sequences.

Spanish molecular biologist Francisco Mojica and colleagues later showed similar structures were present in other organisms and proposed to call them CRISPR: Clustered Regularly Interspaced Short Palindromic Repeats.

In 2005, Mojica and other groups reported the short sequences (or spacers) interrupting the repeats were derived from other DNA belonging to viruses.

Evolutionary biologists Kira Makarova, Eugene Koonin and colleagues eventually proposed CRISPR and the associated Cas9 genes were acting as the immune mechanism. This was experimentally confirmed in 2007 by Rodolphe Barrangou and colleagues.

The CRISPR-associated genes, Cas9, encode a protein that cuts DNA. This is the active part of the defence against viruses, as it destroys the invading DNA.

In 2012, Charpentier and Doudna showed the spacers acted as markers that guided where Cas9 would make a cut in the DNA. They also showed an artificial Cas9 system could be programmed to target any DNA sequence in a lab setting.

This was a groundbreaking discovery which opened the door for CRISPRs wider applications in research.

In 2013, for the first time, groups led by American biochemist Feng Zhang and geneticist George Church reported genome editing in human cell cultures using CRISPR-Cas9. It has since been used in countless organisms from yeast to cows, plants and corals.

Today, CRISPR is the preferred gene-editing tool for thousands of researchers.

Humans have altered the genomes of species for thousands of years. Initially, this was through approaches such as selective breeding.

However, genetic engineering the direct manipulation of DNA by humans outside of breeding and mutations has only existed since the 1970s.

CRISPR-based systems fundamentally changed this field, as they allow for genomes to be edited in living organisms cheaply, with ease and with extreme precision.

CRISPR is currently making a huge impact in health. There are clinical trials on its use for blood disorders such as sickle cell disease or beta-thalassemia, for the treatment of the most common cause of inherited childhood blindness (Leber congenital amaurosis) and for cancer immunotherapy.

CRISPR also has great potential in food production. It can be used to improve crop quality, yield, disease resistance and herbicide resistance.

Used on livestock, it can lead to better disease resistance, increased animal welfare and improved productive traits that is, animals producing more meat, milk or high-quality wool.

A number of challenges to the technology remain, however. Some are technical, such as the risk of off-target modifications (which happen when Cas9 cuts at unintended locations in the genome).

Other problems are societal. CRISPR was famously used in one of the most controversial experiments of recent years.

Read more:Why we need a global citizens assembly on gene editing

Chinese biophysicist He Jiankui unsuccessfully attempted to use the technology to modify human embryos and make them resistant to HIV (human immunodeficiency virus). This led to the birth of twins Lulu and Nana.

We need a broad and inclusive discussion on the regulation of such technologies especially given their vast applications and potential.

To quote CRISPR researcher Fyodor Urnov, Charpentier and Doudnas work really has changed everything.

Dimitri Perrin, Senior Lecturer, Queensland University of Technology

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

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What is CRISPR, the gene editing technology that won the Chemistry Nobel prize? - Grain Central

As we know, COVID-19 is causing large scale loss of life and severe human suffering. With the pandemic spreading across the globe, researchers are racing against the clock to develop diagnostic tools, vaccines and treatments. Recently, WHO has launched a Solidarity clinical trial to assess relative effectiveness of four potential drugs against COVID-19. Further, there are close to 40 clinical trials of vaccines are ongoing, however, as per experts, it may take more than a year to develop a vaccine.

In order to enhance COVID-19 drug discovery and develop rapid testing kits, various academic institutes, non-profit institutes, scientific pioneers and biopharmaceutical companies have also been leveraging benefits of CRISPR technology.

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CRISPR Can be a Solution to Address the COVID-19 Pandemic

The CRISPR / Cas9 system has revolutionized the field of genetic engineering. It enables researchers to alter the genomes of a range of organisms with relative ease. Currently, it has emerged as a promising tool that is used extensively for editing genomes and for the development of novel treatment options. CRISPR is popularly known as search engine for biology, as it has emerged as a location finder, rather than site specific cleavage tool. The figure below highlights the key potential areas and benefits of CRISPR in order to fight against novel coronavirus.

Rapid and Economical Diagnostic Tests

Presently, COVID-19 testing capacity is limited by a number of factors, such as requirements for complex procedures, need for laboratory instrumentation, and dependence on limited supplies. Therefore, there is an urgent need for rapid detection kits. CRISPR has been explored by scientists for diagnosis of infectious diseases. The underlying mechanism involves binding of guide RNA with a protein of Cas family which cuts the target and shreds the nearby RNA or DNA. When CRISPR hits a target, the reporter molecule releases a fluorescent signal. This is further analysed by paper tests dipped into a patient sample, such as blood, urine, or saliva, which further shows up as a line on the testing strip. Researchers have been utilizing CRISPR-based tools and technologies to detect RNA of virus in patient samples. Sherlock Biosciences has already made history, as it received Emergency Use Authorization (EUA) from the US Food and Drug Administration (FDA) for its Sherlock CRISPR SARS-CoV-2 kit for the detection of the virus that causes COVID-19. The kit is designed for use in laboratories and can provide results within an hour. The company claims that more than 1 million tests can be performed within a week.

Enhancing Drug Discovery

CRISPR technology aids in the study of interaction of virus with human cells. This enables the generation of appropriate cell models for faster discovery of new potential treatment options, or identification of an existing drug combination that may provide a treatment solution. For instance, researchers are exploring molecular mechanisms of the novel virus by utilizing CRISPR technology, which can ultimately assist in identifying potential drug combinations.

CRISPR-based COVID-19 Therapy

Researchers at Stanford University have been working on the development of a gene targeting anti-viral agent against COVID-19, using PAC-MAN technology. The technology has been modified to be used against the deadly virus. It consists of a virus-killing enzyme, such as Cas13 and a guide RNA, which commands Cas13 to destroy specific nucleotide sequences in the coronaviruss genome. Based on several studies, it has been revealed that PAC-MAN has the ability to neutralize the coronavirus and stop it from replicating inside cells. Based on information available, work is currently ongoing, and researchers are finding a solution to deliver this technology to lung cells. Multiple delivery methods are currently under evaluation.

A lot of companies are currently active in providing CRISPR-based genome engineering services. To get a detailed information on the key players, recent developments, and the likely market evolution.

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CRISPR Can be a Solution to Address the COVID-19 Pandemic

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CRISPR Can be a Solution to Address the COVID-19 Pandemic | Roots Analysis - Eurowire

In a report released today, Raju Prasad from William Blair maintained a Buy rating on Crispr Therapeutics AG (CRSP). The companys shares closed last Wednesday at $94.30.

According to TipRanks.com, Prasad is a 5-star analyst with an average return of 13.4% and a 53.9% success rate. Prasad covers the Healthcare sector, focusing on stocks such as Global Blood Therapeutics, Alexion Pharmaceuticals, and Rocket Pharmaceuticals.

Currently, the analyst consensus on Crispr Therapeutics AG is a Strong Buy with an average price target of $95.88, implying a 5.1% upside from current levels. In a report released today, Needham also assigned a Buy rating to the stock with a $105.00 price target.

See todays analyst top recommended stocks >>

The company has a one-year high of $111.90 and a one-year low of $32.30. Currently, Crispr Therapeutics AG has an average volume of 870.1K.

Based on the recent corporate insider activity of 41 insiders, corporate insider sentiment is neutral on the stock.

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

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

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William Blair Sticks to Its Buy Rating for Crispr Therapeutics AG (CRSP) - Smarter Analyst

CRISPR And CRISPR-Associated (Cas) Genes Market report would come handy to understand the competitors in the market and give an insight into sales, volumes, revenues in the CRISPR And CRISPR-Associated (Cas) Genes Industry & will also assists in making strategic decisions. The report also helps to decide corporate product & marketing strategies. It reduces the risks involved in making decisions as well as strategies for companies and individuals interested in the CRISPR And CRISPR-Associated (Cas) Genes industry. Both established and new players in CRISPR And CRISPR-Associated (Cas) Genes industries can use the report to understand the CRISPR And CRISPR-Associated (Cas) Genes market.

In Global Market, the Following Companies Are Covered:

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Analysis of the Market:

Clustered regularly interspaced short palindromic repeats (CRISPR) are segments of prokaryotic DNA containing short repetitions of base sequences. Each repetition is followed by short segments of spacer DNA from previous exposures to a bacteriophage virus or plasmid.

The CRISPR/Cas system is a prokaryotic immune system that confers resistance to foreign genetic elements such as those present within plasmids and phages, and provides a form of acquired immunity. CRISPR associated proteins (Cas) use the CRISPR spacers to recognize and cut these exogenous genetic elements in a manner analogous to RNA interference in eukaryotic organisms. CRISPRs are found in approximately 40% of sequenced bacterial genomes and 90% of sequenced archaea.

CRISPR And CRISPR-Associated (Cas) Genes industry is relatively concentrated, manufacturers are mostly in the Europe and North America. Among them, North America region accounted for more than 45.70% of the total market of global CRISPR And CRISPR-Associated (Cas) Genes.

The global CRISPR And CRISPR-Associated (Cas) Genes market is valued at 713.8 million USD in 2020 is expected to reach 7696.7 million USD by the end of 2026, growing at a CAGR of 40.0% during 2021-2026.

This report focuses on CRISPR And CRISPR-Associated (Cas) Genes volume and value at the global level, regional level and company level. From a global perspective, this report represents overall CRISPR And CRISPR-Associated (Cas) Genes market size by analysing historical data and future prospect. Regionally, this report focuses on several key regions: North America, Europe, China and Japan et

CRISPR And CRISPR-Associated (Cas) Genes Market Breakdown by Types:

CRISPR And CRISPR-Associated (Cas) Genes Market Breakdown by Application:

Critical highlights covered in the Global CRISPR And CRISPR-Associated (Cas) Genes market include:

The information available in the CRISPR And CRISPR-Associated (Cas) Genes Market report is segmented for proper understanding. The Table of contents contains Market outline, Market characteristics, Market segmentation analysis, Market sizing, customer landscape & Regional landscape. For further improving the understand ability various exhibits (Tabular Data & Pie Charts) has also been used in the CRISPR And CRISPR-Associated (Cas) Genes Market report.

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In the end, CRISPR And CRISPR-Associated (Cas) Genes Industry report provides the main region, market conditions with the product price,profit, capacity, production, supply, demand and market growth rateand forecast etc. This report also Present newproject SWOT analysis,investment feasibility analysis, andinvestment return analysis.

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CRISPR And CRISPR-Associated (Cas) Genes Market 2020 to Flourish with an Impressive CAGR of XX% in the year 2026, Market Size & Growth with...

Report is a detailed study of the CRISPR and CAS Gene market, which covers all the essential information required by a new market entrant as well as the existing players to gain a deeper understanding of the market. The primary objective of this research report named CRISPR and CAS Gene market is to help making reliable strategic decisions regarding the opportunities in CRISPR and CAS Gene market.

Major Market Players with an in-depth analysis:

Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., 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.

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The Global CRISPR and CAS Gene Market segments and Market Data Break Down are illuminated below:

By Product Type:Vector-based CasDNA-free CasGlobal CRISPR and CAS Gene Market, By Application:Genome EngineeringDisease modelsFunctional GenomicsKnockdown/activationOther Applications

The CRISPR and CAS Gene market report offers the current state of the market around the world. The report starts with the market outline and key of the CRISPR and CAS Gene market which assumes a significant job for clients to settle on the business choice. It additionally offers the key focuses to upgrade the development in the CRISPR and CAS Gene market. Some fundamental ideas are likewise secured by reports, for example, item definition, its application, industry esteem chain structure and division which help the client to break down the market without any problem. Also, the report covers different factors, for example, arrangements, efficient and innovative which are affecting the CRISPR and CAS Gene business and market elements.

Download Sample Report of CRISPR and CAS Gene Market Report 2020 (Coronavirus Impact Analysis on CRISPR and CAS Gene Market)

Competitive Analysis has been done to understand overall market which will be helpful to take decisions. Major players involved in the manufacture of CRISPR and CAS Gene product has been completely profiled along with their SWOT. Some of the key players include Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., 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. It helps in understanding their strategy and activities. Business strategy described for every company helps to get idea about the current trends of company. The industry intelligence study of the CRISPR and CAS Gene market covers the estimation size of the market each in phrases of value (Mn/Bn USD) and volume (tons). Report involves detailed chapter on COVID 19 and its impact on this market. Additionally, it involves changing consumer behavior due to outbreak of COVID 19.

Further, report consists of Porters Five Forces and BCG matrix as well as product life cycle to help you in taking wise decisions. Additionally, this report covers the inside and out factual examination and the market elements and requests which give an entire situation of the business.

Regional Analysis for CRISPR and CAS Gene

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Chapters Define in TOC (Table of Content) of the Report:

Chapter 1: Market Overview, Drivers, Restraints and Opportunities, Segmentation

Overview

Chapter 2: COVID Impact

Chapter 3: Market Competition by Manufacturers

Chapter 4: Production by Regions

Chapter 5: Consumption by Regions

Chapter 6: Production, By Types, Revenue and Market share by Types

Chapter 7: Consumption, By Applications, Market share (%) and Growth Rate by

Applications

Chapter 8: Complete profiling and analysis of Manufacturers

Chapter 9: Manufacturing cost analysis, Raw materials analysis, Region-wise

Manufacturing expenses

Chapter 10: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 11: Marketing Strategy Analysis, Distributors/Traders

Chapter 12: Market Effect Factors Analysis

Chapter 13: Market Forecast

Chapter 14: CRISPR and CAS Gene Research Findings and Conclusion, Appendix, methodology and data source

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The qualitative contents for geographical analysis will cover market trends in each region and country which includes highlights of the key players operating in the respective region/country, PEST analysis of each region which includes political, economic, social and technological factors influencing the growth of the market. The research report includes specific segments by Type and by Application. This study provides information about the sales and revenue during the historic and forecasted period of 2020 to 2027.

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Covid-19 Impact On CRISPR and CAS Gene Market Study: An Emerging Hint of Opportunity Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC,...