Summary of our Crispr action

Summary of our Crispr action

After the Crispr snack of 5 March 2019, the Belgian Food Safety Authority questioned Science for Democracy’s coordinators Marco Cappato and Marco Perduca for over three hours. While there has been no notification of any sanction yet (criminal or administrative), the Agency has sent a recap file, which includes the first report, the list of confiscated material, the audition report and pictures of the event taken by agents in front of the European Parliament. Enjoy the reading (in French) and, should you wish to support Science for Democracy, here is how you can donate! Thank you!

Scientists use editing tool to identify key cancer genes

Scientists use editing tool to identify key cancer genes

Hopes of new treatments after research uncovers genes essential to disease’s survival

Researchers working with a revolutionary gene editing tool have discovered thousands of genes that are essential for the survival of cancer cells, holding out the prospect of major advances in treatment.

Scientists from the Wellcome Sanger Institute in Cambridgeshire worked with the Crispr/Cas9 system to disrupt every gene within 30 different types of cancer.

This led them to identifying 600 genes that could be used in precision treatments that would mean sufferers not having to endure the side effects of options such as chemotherapy and radiotherapy.

One gene identified is Werner syndrome RecQ helicase, which researchers found was essential for keeping alive some of the most unstable cancers but which cannot currently be targeted.

The research, which was a collaboration between the Wellcome Sanger Institute, the European Bioinformatics Institute and the pharmaceutical company GlaxoSmithKline, was published in the journal Nature on Wednesday.

It could help to bring down the cost of making effective cancer treatments: the institute said that it currently costs more than $1bn (£760m) to make a single drug, and that 90% of these fail during testing and development.

Praising the tool that made the breakthrough possible, Dr Kosuke Yusa, the co-lead author of the findings, said Crispr was “incredibly powerful” and “enables us to do science at a scale and with a precision that we couldn’t do five years ago.

“With Crispr we have discovered a very exciting opportunity to develop new drugs targeting cancers.”

Dr Mathew Garnett, also co-lead author, said: “The Cancer Dependency Map is a huge effort to identify all the weaknesses that exist in different cancers so we can use this information to empower the next generation of precision cancer treatments.

“Ultimately we hope this impacts on the way we treat patients, so many more patients get effective therapies.”

Prof Karen Vousden, Cancer Research UK’s chief scientist, told the BBC: “What makes this research so powerful is the scale.

“This work provides some excellent starting points and the next step will be a thorough analysis of the genes that have been identified as weaknesses in this study, to determine if they will one day lead to the development of new treatments for patients.”

 

Credit: The Guardian

Gene-edited foods are safe, Japanese panel concludes

Gene-edited foods are safe, Japanese panel concludes

Japan will allow gene-edited foodstuffs to be sold to consumers without safety evaluations as long as the techniques involved meet certain criteria, if recommendations agreed on by an advisory panel yesterday are adopted by the Ministry of Health, Labour and Welfare. This would open the door to using CRISPR and other techniques on plants and animals intended for human consumption in the country.

“There is little difference between traditional breeding methods and gene editing in terms of safety,” Hirohito Sone, an endocrinologist at Niigata University who chaired the expert panel, told NHK, Japan’s national public broadcaster.

How to regulate gene-edited food is a hotly debated issue internationally. Scientists and regulators have recognized a difference between genetic modification, which typically involves transferring a gene from one organism to another, and gene editing, in which certain genes within an organism are disabled or altered using new techniques such as CRISPR. That’s why a year ago, the U.S.Department of Agriculture concluded that most gene-edited foods would not need regulation. But the European Union’s Court of Justice ruled in July 2018 that gene-edited crops must go through the same lengthy approval process as traditional transgenic plants.

Now, Japan appears set to follow the U.S. example. The final report, approved yesterday, was not immediately available, but an earlier draft was posted on the ministry website. The report says no safety screening should be required provided the techniques used do not leave foreign genes or parts of genes in the target organism. In light of that objective, the panel concluded it would be reasonable to require information on the editing technique, the genes targeted for modification, and other details from developers or users that would be made public while respecting proprietary information.

The recommendations leave open the possibility of requiring safety evaluations if there are insufficient details on the editing technique. The draft report does not directly tackle the issue of whether such foods should be labeled. The ministry is expected to largely follow the recommendations in finalizing a policy on gene-edited foods later this year.

Consumer groups had voiced opposition to the draft recommendations, which were released for public comment in December 2018. Using the slogan “No need for genetically modified food!” the Consumers Union of Japan joined other groups circulating a petition calling for regulating the cultivation of all gene-edited crops, and safety reviews and labeling of all gene-edited foods.

Whether consumers will embrace the new technology remains to be seen. Japan has approved the sale of genetically modified (GM) foods that have passed safety tests as long as they are labeled. But public wariness has limited consumption and has led most Japanese farmers to shun GM crops. The country does import sizable volumes of GM processed food and livestock feed, however. Japanese researchers are reportedly working on gene-edited potatoes, tomatoes, rice, chicken, and fish. “Thorough explanations [of the new technologies] are needed to ease public concerns,” Sone said.

 

Credit: Science Magazine

 

Commissioner Andriukaitis responds to our CRISPR snack

Commissioner Andriukaitis responds to our CRISPR snack

Read the European Commission’s Response

Further to our March 5th CRISPR snack, the Director for food and feed safety, innovation in the Directorate-General for Health and Food Safety of the European Commission, Sabine Jülicher, responded to Science for Democracy with a letter, on behalf of Commissioner Andriukaitis. In it, the European Commission restates that enforcement of regulation is a national competence, but acknowledges the importance of stakeholder engagement and thanks Science for Democracy for informing the Commissioner about our initiatives.

 

What was asked on the 5 March CRISPR snack

What was asked on the 5 March CRISPR snack

On 5 March 2019, Marco Cappato, treasurer of the Luca Coscioni Association and Marco Perduca, coordinator of Science for Democracy, organized a demonstration in front of the European Parliament in Brussels to denounce the serious repercussions in terms of limitation to scientific research of a judgment of the European Court of Justice adopted on 25 July 2018.

In conclusion of the public event participants ate some home-made rice pudding prepared with genetically edited rice with the CRISPR technique. During the snack the Belgian authorities confiscated the rice, identifying Marco Cappato and Marco Perduca and summoning them on 2 April to justify their actions and explain the origin of the rice.

On 2 April 2019, Perduca and Cappato, accompanied by Guido Long, were heard for almost three hours by officials of the Federal Agency for Food Chain Security.

 

Below what was asked to Mr. Perduca

 

Marco PERDUCA declares to act on behalf of the non-profit association “Science for Democracy” as an administrator, is present without having received a written convocation with a list of rights and is asked questions in the presence of Mr. CAPPATO.

Questions are asked on the following:

  1. Distribution of food products manufactured from unauthorized GMO rice;
  2. non-respect of the seizure of the aforementioned food product.

Agency inspectors inform Mr. PERDUCA:

1 – that he has the right, prior to interrogation, to consult with a lawyer of his choice or with a lawyer assigned to him, and who has the possibility of being assisted by him during the interrogation; that he must take the necessary measures himself to be assisted by a lawyer and that he can request to have the hearing postponed only once for this purpose;

2- that he has the possibility, after having identified himself, to make a declaration, to answer the questions that will be addressed to him or to remain silent;

3- that he cannot be forced to accuse himself;

4- that his statements can be used as evidence by justice and can therefore be used against him or against another person;

5- that he can request that all questions that are addressed to him as well as the answers are listed in the terms used;

6 – that he is not deprived of freedom and that he can come and go as he wishes;

7- that he may request to be subjected to certain information acts or hearings;

8- that he can use the documents in his possession, without this causing the postponement of the interrogation and that he can, during the interrogation or subsequently, request that they be attached to the report or file;

9- that he can obtain a copy of the interrogation report free of charge.

Mr. PERDUCA is given a written declaration of his rights to the interested party before the first interrogation.

Mr. PERDUCA declares to renounce his right to a preliminary consultation with a lawyer and to be assisted by him during the audition. The interested party signs a waiver form dated and attached to the hearing report.

The questioned person lets us know that he wants to express himself in French and that he chooses French as the language of justice.

 

Audition:

 

I come today to your offices according to your desire to proceed with my questioning as part of your check of 5 March 2019 made in front of the European Parliament at the Place du Luxembourg in Ixelles on the occasion of our event “Give CRISPR a chance”.

You ask me if our ASBL pays its members. I answer you no.

You ask me how many members our ASBL has. I can’t tell you a precise number. I would say about fifty members of which only a part was present at our event. Our members come from all horizons including non-EU countries.

You ask me what is the calendar of our events. I answer you that our event of March 5 was aimed at keeping the public and MEPs informed about the decision of the Luxembourg Court of July 25, 2018, and to explain what CRISPR is as a new technology. In the past, we organized a picnic in Rome on 17 September 2018 in front of the parliament and on 4 October in front of the university in Milan. On 8 October 2018 we organized a debate on the right to science at the United Nations in Geneva. On March 21, 2019 we held a debate on human rights and scientific research on drugs in Vienna. On March 19, 2019, a debate on biotechnology in agriculture in the Senate in Rome. On February 20 we organized a debate on science and innovation in Italy in Rome. We have sent a series of recommendations to the United Nations following these debates. All our past events are listed on our website www.sciencefordemocracy.org on the “when” page. On 8 April we have a seminar on environmental policy assessment in London. On 5 July we will organize a debate in Rome on the critical aspects of science and research in Italy. A debate on science is planned for October in Geneva. In 2020, an international congress in Ethiopia. We have not received any restriction regarding our events, nor during the picnics organized in Italy. Members of parliament are partners in our projects. You ask me if we have an upcoming event about CRISPR and I answer no.

You ask me why we do not consider the CRISPR method as a scientific method assimilated to a genetic modification. The answer is simple: because this does not correspond to the contents of the 2001 Directive. In particular, these are genetic modifications invented in 2012 that are not distinguishable from a natural modification. GMOs are modifications of a whole series of genes. The CRISPR-cas method cuts the DNA sequence without any introduction of genome from other species. CRISPR allows us to decide when genome modification will take place instead of depending on chance as it happens in nature.

You ask me if we know the role of the AFSCA. I answer you that before your action I did not know the AFSCA. You inform me that the AFSCA is based on established regulation.

We want to give scientists the chance to do their job. You ask me why we organized this event in front of Parliament. I reply that it is the European Parliament that must change the European Directive of 2001. Why have you associated the distribution of genetically modified rice with the public during your awareness-raising event? I reply that it is a question of trust in the products we and scientists defend. We would like to clarify that we do not agree with the notion of placing on the market because our militants wore a specific t-shirt with the words “Give CRISPR a chance”. This visual differentiation was useful for us to direct the distribution only towards our sympathizers. Our supporters are not all members who paid the registration fees but appear on our mailing list when we organize events. We also want to clarify that we dispute the genetically modified nature of the proposed rice because it is currently impossible to technologically determine that genetic modifications are the result of human intervention rather than a natural mechanism. The CRISPR method is not an expansive modification of the genome.

We did not respond favourably to your injunction not to distribute the product because it was important for us to emphasize our confidence in technology by making our sympathizers taste the product and consuming it ourselves. As for rice, you ask me where the preparation was made. I answer you that the preparation of the rice was made by us at the residence of some friends. I do not wish to give you the address of this kitchen.

You ask me where the rice that was part of the preparation comes from. I reply that we asked for this modified CRISPR rice in Italy to a scientist whose name I will not give. In Japan there is more advanced research on CRISPR-cas on rice. That is where the rice was produced. I cannot tell you what status CRISPR-cas rice has in Japan. Rice arrives by plane from Japan to Rome. I import the rice myself as a physical person. My offices were in Rome at Via di Torre Argentina 76, 00186. I worked there as a consultant in the field of human rights.

You ask me if any derogation application has been introduced. We do not know the conditions that would allow us to consider such a situation. About two years ago, before creating our ASBL, we were already interested in the CRISPR-cas method.

The hearing took place in accordance with the questions and answers, according to the terms used.

We read the hearing report to the interviewee, after which he confirmed he did not want to add or modify anything.

The interviewee received a copy of his hearing and signature below for receipt.

The hearing ends on 2 April 2019 at 3.35pm.

 

Thanks to Guido Long for the translation from French.

 

Artificial Intelligence In Humanoid Robots

Artificial Intelligence In Humanoid Robots

When people think of Artificial Intelligence (AI), the major image that pops up in their heads is that of a robot gliding around and giving mechanical replies. There are many forms of AI but humanoid robots are one of the most popular forms. They have been depicted in several Hollywood movies and if you are a fan of science fiction, you might have come across a few humanoids. One of the earliest forms of humanoids was created in 1495 by Leonardo Da Vinci. It was an armor suit and it could perform a lot of human functions such as sitting, standing and walking. It even moved as though a real human was inside it.

Initially, the major aim of AI for humanoids was for research purposes. They were being used for research on how to create better prosthetics for humans. Now, humanoids are being created for several purposes that are not limited to research. Modern-day humanoids are developed to carry out different human tasks and occupy different roles in the employment sector. Some of the roles they could occupy are the role of a personal assistant, receptionist, front desk officer and so on.

The process of inventing a humanoid is quite complex and a lot of work and research is put into the process. Most times, inventors and engineers face some challenges. First-grade sensors and actuators are very important and a tiny mistake could result in glitching. Humanoids move, talk and carry out actions through certain features such as sensors and actuators.

People assume that humanoid robots are robots that are structurally similar to human beings. That is, they have a head, torso, arms and legs. However, this is not always the case as some humanoids do not completely resemble humans.

Some are modeled after only some specific human parts such as the human head. Humanoids are usually either Androids or Gynoids. An Android is a humanoid robot designed to resemble a male human while gynoids look like female humans.

Humanoids work through certain features. They have sensors that aid them in sensing their environments. Some have cameras that enable them to see clearly. Motors placed at strategic points are what guide them in moving and making gestures. These motors are usually referred to as actuators.

A lot of work, finances and research are put into making these humanoid robots. The human body is studied and examined first to get a clear picture of what is about to be imitated. Then, one has to determine the task or purpose the humanoid is being created for. Humanoid robots are created for several purposes. Some are created strictly for experimental or research purposes. Others are created for entertainment purposes. Some humanoids are created to carry out specific tasks such as the tasks of a personal assistant using AI, helping out at elderly homes, and so on.

The next step scientists and inventors have to take before a fully functional humanoid is ready is creating mechanisms similar to human body parts and testing them. Then, they have to go through the coding process which is one of the most vital stages in creating a humanoid. Coding is the stage whereby these inventors program the instructions and codes that would enable the humanoid to carry out its functions and give answers when asked a question.

Doesn’t sound so difficult, right? However, it would be foolhardy to think that creating a humanoid is as easy as creating a kite or a slingshot in your backyard. Although humanoid robots are becoming very popular, inventors face a few challenges in creating fully functional and realistic ones. Some of these challenges include:
  • Actuators: These are the motors that help in motion and making gestures. The human body is dynamic. You can easily pick up a rock, toss it across the street, spin seven times and do the waltz. All these can happen in the space of ten to fifteen seconds. To make a humanoid robot, you need strong, efficient actuators that can imitate these actions flexibly and within the same time frame or even less. The actuators should be efficient enough to carry a wide range of actions.
  • Sensors: These are what help the humanoids to sense their environment. Humanoids need all the human senses: touch, smell, sight, hearing and balance to function properly. The hearing sensor is important for the humanoid to hear instructions, decipher them and carry them out. The touch sensor prevents it from bumping into things and causing self-damage. The humanoid needs a sensor to balance movement and equally needs heat and pain sensors to know when it faces harm or is being damaged. Facial sensors also need to be intact for the humanoid to make facial expressions, and these sensors should be able to carry a wide range of expressions.

Making sure that these sensors are available and efficient is a hard task:

  • AI-based Interaction: The level at which humanoid robots can interact with humans is quite limited. This where Artificial Intelligence is critical. It can help decipher commands, questions, statements and might even be able to give witty, sarcastic replies and understand random, ambiguous human ramblings.

However, some humanoid robots are so human-like and efficient, that they have become quite popular. Here are a few of them.

  1. Sophia: This is the world’s first robot citizen. She was introduced to the United Nations on October 11, 2017. On October 25th, she was granted Saudi Arabian citizenship, making her the first humanoid robot ever to have a nationality.

Sophia was created by Hanson robotics and can carry out a wide range of human actions. It is said that she is capable of making up to fifty facial expressions and can equally express feelings. She has very expressive eyes and her Artificial Intelligence revolves around human values. She has an equal sense of humor. This particular humanoid was designed to look like the late British actress, Audrey Hepburn. Since she was granted citizenship, Sophia has attended several interviews, conferences and is now one of the world’s most popular humanoids.

  1. The Kodomoroid TV Presenter: This humanoid robot was invented in Japan. Her name is derived from the Japanese word for child- Kodomo- and the word ‘Android’. She speaks a number of languages and is capable of reading the news and giving weather forecasts.

She has been placed at the Museum of Emerging Science and Innovation in Tokyo where she currently works.

  1. Jia Jia: This humanoid robot was worked on for three years by a team at the University of Science and Technology of China before its release. She is capable of making conversations but has limited motion and stilted speech. She does not have a full range of expressions but the team of inventors plans to make further developments and infuse learning abilities in her. Although her speech and vocabulary need further work, she is still fairly realistic.

Humanoid robots are here to stay and over time, with AI making progress, we might soon find them everywhere in our daily lives.

Open-access pioneer Randy Schekman on Plan S and disrupting scientific publishing

Open-access pioneer Randy Schekman on Plan S and disrupting scientific publishing

Nobel laureate Randy Schekman shook up the publishing industry when he launched the open-access journal eLife in 2012.

Armed with millions in funding from three of the world’s largest private biomedical charities — the Wellcome Trust, the Max Planck Society and the Howard Hughes Medical Institute — Schekman designed the journal to compete with publishing powerhouses such as NatureScience and Cell. (Nature’s news team is independent of its journal team and its publisher, Springer Nature.)

eLife experimented with innovative approaches such as collaborative peer review — in which reviewers work together to vet research — that caused ripples in scientific publishing.

And for the first few years, researchers could publish their work in eLifefor free. That came to an end in 2017, because the journal needed more revenue streams to help it to grow.

Schekman stepped down from eLife on 31 January to chair an advisory council for the Aligning Science Across Parkinson’s initiative, funded by the Sergey Brin Family Foundation in San Francisco, California, to better coordinate research into Parkinson’s disease.

Nature’s news team asked Schekman about the impact he thinks eLifehas had on scholarly publishing, and about the future of open-access journals.

eLife?

eLife started with a quite substantial dowry from the Wellcome Trust, the Max Planck Society and the Howard Hughes Medical Institute. It was a clean slate, and we could do more or less what we wanted as long as it was successful. Success was not defined by impact factor — we were firmly committed to dampening its influence in science. It was defined by the kinds of papers that people send to us for publication: we would judge it a success if our own board members sent us their best work to publish — some have and some not yet.

One of the principal decisions we made was that all choices of which papers to include should come from working scientists. I took advantage of my experiences as editor-in-chief from 2006–11 at the Proceedings of the National Academy of Sciences (PNAS) to cook up this idea of reviewers consulting each other when evaluating a paper. This has become a unique feature of eLife.

What are the benefits and drawbacks of eLife’s practice of collaborative peer review?

There are many benefits. When you agree to review a paper for eLife, you know that your identity will be shared with other reviewers, so you can’t hide behind your anonymity. People can’t say things that they can’t defend. When a paper is considered appropriate for revision, the board member who is monitoring the review often writes the letter to the author. They organize it so that only the key points are written in a summary, which is helpful for the authors.

One downside is that it creates a bit more work, because the reviewers are not done when they submit their reviews. It takes time to have that conversation to craft a decision letter. The reviewers tell me that they enjoy this process.

Another potential downside is that you can have an assistant professor whose opinion is going against that of an established person in their field — and the assistant professor might not say what they think. But I’ve only had one reviewer tell me they felt intimidated during the process. My feeling is that it might be quite the opposite: the younger person is closer to the technical aspects of the work, and in many cases, young people who agree to review a paper want to prove themselves. If it were a problem, we would find it difficult to get young people to serve as reviewers, and we don’t.

What do you think of Plan S, an initiative to make all papers open access on publication?

I’m very supportive of this. Open access is the future. Commercial journals have been fighting against this very hard because it poses a clear danger to their profit margin. The public has paid for this research, so they deserve to have access to it.

However, there are legitimate expenses of publishing that need to be made clear. PNAS is published by a science society that does not make a profit, and it recently said that it estimates the cost of publishing a paper to be US$6,000, so any cap on article-processing charges that could come from Plan S might have to allow for this. But $6,000 seems very high to me. It might be that journals may have different article-processing charges depending on their selectivity.

How do you think Plan S will affect scholarly publishing?

There will be a shakedown in the business. Some journals will lose out. Publishing is not a static business — the advent of the preprint server has really changed things, for example. Journals are going to change, and Plan S could have a strong influence.

Do you think that eLife can survive in a Plan S world without extra funding?

The journal still receives income from charitable funders, as well as from article-processing charges. But we hope for eLife to be self-sustaining within two or three years. The financial models we have drawn show that it can be done. We want to keep having the same high standards and peer-review mechanisms going forward. We believe that eLife has the bandwidth to grow maybe two-fold in submissions — and if we do this, we can sustain ourselves without charitable funding.
Deal reveals what scientists in Germany are paying for open access

Deal reveals what scientists in Germany are paying for open access

Project Deal, a consortium of libraries, universities, and research institutes in Germany, has unveiled an unprecedented deal with a major journal publisher—Wiley—that is drawing close scrutiny from advocates of open access to scientific papers.

The pact, signed last month but made public this week, has been hailed as the first such country-wide agreement within a leading research nation. (Only institutions in the United States, China, and the United Kingdom publish more papers.) It gives researchers working at more than 700 Project Deal institutions access to the more than 1500 journals published by Wiley, based in Hoboken, New Jersey, as well as the publisher’s archive. It also allows researchers to make papers they publish with Wiley free to the public at no extra cost.

This business arrangement, known as a “publish and read” deal, has been touted as one way to promote open-access publishing. But until this week, a key part of the Wiley agreement—how much it will cost—had been secret.

Now, the numbers are out. Germany will pay Wiley €2750 for each paper published in one of the publisher’s so-called hybrid journals, which contain both paywalled and free papers. The contract anticipates researchers will publish about 9500 such papers per year, at a cost of €26 million. In addition, researchers will get a 20% discount on the price of publishing in Wiley journals that are already open access.

The deal is an important step toward more open access in scientific publishing, but the per paper fee of €2750 seems high, says Leo Waaijers, an open-access advocate and retired librarian at the Delft University of Technology in the Netherlands. Dutch researchers are paying Wiley just €1600 per paper under a similar deal in the Netherlands, he notes. “It’s the same process, the same product, so why the price difference?” he says.

The explanation is that Germany’s deal with Wiley was designed to be “more or less budget-neutral,” says Gerard Meijer, a physicist at the Fritz Haber Institute, part of the Max Planck Society in Berlin, and one of the negotiators for Project Deal. The goal was to keep Germany’s 2019 payments to Wiley about the same as they were in 2018, he says. And as a larger country with more institutions, Germany paid more in subscription fees to Wiley than the Netherlands. That translated to a higher article publishing fee. But the difference is that papers from Project Deal researchers will now be freely available around the world. In addition, some institutions have gained access to journals that they did not have access to before.

One advantage of the deal is that German researchers will no longer be paying twice for Wiley’s hybrid journals—once for a subscription, and again if they want to make a paper free—says Lidia Borrell-Damian of the European University Association in Brussels. “Germany seems protected from double-dipping … and that’s important,” she says.

Eventually, Waaijers hopes German institutions will be able to negotiate lower open-access publishing fees. But he sees the current contract, which runs for 3 years, as a good first step. “I think it is not possible for Germany to say to Wiley at the moment: ‘We want a contract for 1600 [euros] per article,’” he says. “That would mean an enormous step back financially for Wiley, and they are absolutely not prepared to make that step.”

The fact that the details of the German contract have become public is also important, Borrell-Damian says. “Contracts should be public because this is about public money spent,” she says. And if other countries sign similar deals, and the details become public, then “the whole game of price comparison may start,” Waaijers says. And that, open-access advocates say, could produce pressure for even lower publishing fees.

Expanding the Gene Editing Toolbox: Scientists Sharpen Their Molecular Scissors

Expanding the Gene Editing Toolbox: Scientists Sharpen Their Molecular Scissors

Wake Forest Institute for Regenerative Medicine (WFIRM) scientists have figured out a better way to deliver a DNA editing tool to shorten the presence of the editor proteins in the cells in what they describe as a “hit and run” approach.

CRISPR (clustered regularly interspaced short palindromic repeats) technology is used to alter DNA sequences and modify gene function. CRISPR/Cas9 is an enzyme that is used like a pair of scissors to cut two strands of DNA at a specific location to add, remove or repair bits of DNA. But CRISPR/Cas9 is not 100 percent accurate and could potentially cut unexpected locations, causing unwanted results.

“One of the major challenges of CRISPR/Cas9 mRNA technologies is the possibility of off-targets which may cause tumors or mutations,” said Baisong Lu, Ph.D, assistant professor of regenerative medicine at WFIRM and one of the lead authors of the paper. Although other types of lentivirus-like bionanoparticles (LVLPs) have been described for delivering proteins or mRNAs, Lu said, “the LVLP we developed has unique features which will make it a useful tool in the expanding genome editing toolbox.”

To address the inaccuracy issue, WFIRM researchers asked the question: Is there a way to efficiently deliver Cas9 activity but achieve transient expression of genome editing proteins? They tested various strategies and then took the best properties of two widely used delivery vehicles – lentivirus vector and nanoparticles – and combined them, creating a system that efficiently packages Cas9 mRNA into LVLPs, enabling transient expression and highly efficient editing.

Lentiviral vector is a widely used gene delivery vehicle in research labs and is already widely used for delivering the CRISPR/Cas9 mRNA technology for efficient genome editing. Nanoparticles are also being used but they are not as efficient in delivery of CRISPR/Cas9.

The WFIRM team published its findings in a paper published recently in the journal Nucleic Acids Research.

“By combining the transient expression feature of nanoparticle-delivery strategies while retaining the transduction efficiency of lentiviral vectors, we have created a system that may be used for packaging various editor protein mRNA for genome editing in a ‘hit and run’ manner,” said Anthony Atala, M.D., director of WFIRM and co-lead author of the paper. “This system will not only improve safety but also avoid possible immune response to the editor proteins, which could improve in vivo gene editing efficiency which will be useful in research and clinical applications.”

This article has been republished from materials provided by Wake Forest Institute for Regenerative Medicine.

Note: material may have been edited for length and content. For further information, please contact the cited source.

Chinese gene-editing scientist He Jiankui may have made the twins smarter, scientists say

Chinese gene-editing scientist He Jiankui may have made the twins smarter, scientists say

The controversial Chinese scientist who shocked the world by claiming he edited the genes of twin sisters may have inadvertently enhanced their brains, scientists say.

He Jiankui announced last November he had used technology known as CRISPR to alter the embryonic genes of twins Lulu and Nana in an attempt to protect them from infection with the HIV virus carried by their father — a move that received widespread international condemnation.

New research published in the journal Cell last Thursday claims the same alteration to the girls’ DNA — deleting a gene called CCR5 — not only makes mice smarter but also improves human brain recovery after a stroke and could be linked to greater success in school, MIT Technology Review reported.

But Alcino J Silva, a neurobiologist at the University of California, Los Angeles (UCLA), whose lab uncovered the major new role for the CCR5 gene in memory and the brain’s ability to form new connections, said the exact effect on the girls’ cognition was impossible to predict.

He added that was “why it should not be done”.

While there was no proof that Mr He aimed to enhance the babies’ intelligence, he told an international human genome editing summit in Hong Kong last November that he was aware of a previous study co-authored by Dr Silva in 2016 that showed removing the CCR5 gene from mice significantly improved their memory.

The team had looked at more than 140 different genetic alterations to find which made mice smarter.

I saw that paper, and I believe it needs more independent verification, he said in a response to a question asking if he had inadvertently enhanced cognitive ability of the gene-edited babies.

Mr He also told the panellists he was “against using genome editing for enhancement”.

Regardless of Mr He’s intentions, the new research forms part of increasing evidence that the CCR5 gene plays a significant role in the brain.

According to MIT Technology Review, the latest paper on CCR5 also showed that people missing at least one copy of the CCR5 gene appear to perform better at school, therefore suggesting a link to everyday intelligence.

Could it be conceivable that at one point in the future we could increase the average IQ of the population? I would not be a scientist if I said no, Dr Silva told MIT Technology Review.

The work in mice demonstrates the answer may be yes. But mice are not people.

We simply don’t know what the consequences will be in mucking around. We are not ready for it yet.

Mr He, who was fired from his role at the Southern University of Science and Technology in Shenzhen, has not been seen in public since last November, shortly after his gene-editing announcement.

He is now believed to be staying in a heavily guarded university-owned apartment in Shenzhen.

Chinese authorities have also put a halt to all forms of research like Mr He’s and ordered universities to review all research work on gene editing.