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First CRISPR children: 6 questions left

The CRISPR-Cas9 rescue tool is loaded in a pipette. Credit: Mark Schiefelbein / AP / Shutterstock The meeting where He…

The CRISPR-Cas9 rescue tool is loaded in a pipette. Credit: Mark Schiefelbein / AP / Shutterstock

The meeting where He Jiankui declared his extraordinary statement to have helped to produce the first babies &#821

1; twin girls – born with edited genomes came to an end with a statement that came down hard by the scientist.

“We heard an unexpected and deeply disturbing statement that human embryos had been edited and implanted, resulting in a pregnancy and the birth of twins,” says the statement released by the organizing committee at the Second International Human Resource Summit in Hong Kong November 29th. “Although the changes have been verified, the procedure was irresponsible and failed to comply with international standards.”

Similar criticisms rained since the revelation earlier this week that he had used CRISPR-Cas9 to modify the CCR5 gene into two embryos, which he then implanted in a woman. The gene encodes a protein that many strains of HIV use to infect immune cells into two embryos, which he then implanted in a woman.

As a researcher doing an analysis of the week’s events, Nature summarizes six major questions that are still unanswered.

first Is he Jiankui in trouble?

On November 27, China’s National Health Ministry called the Guangdong government, where he is a university, the Southern University of Science is – to investigate Han. Two days later, the Ministry of Science ordered that he stopped doing science, he had already said that the experiments were in operation. How the Guangdong investigation will continue to be unclear. He is accused of violating a guideline for the Ministry of Health from 2003, which is not a law and has no clear sanctions.

If he is a university, Southern University of Science and Technology, will take some action against him is also unclear. A spokesman talked about Nature that he “can not reveal such information at this time” and to wait for official statements “at an appropriate time”. He has been in leave since February 2018 and is scheduled to be resigned until January 2021; This week, the University criticized its claims and spaced from its work.

On November 27th, the laboratory hosted the university – as he has referred to for information about the refurbished infants – the place for his lab rest. Several statements promising Han Jiankui’s achievements have also disappeared from government websites. A post on the website of the Ministry of Science, which describes a genomic sequencing technique that he developed and a post that promises Han genomic sequencing technology on the Thousand Talent Plan website – a prestigious system for bringing leading academics back to China – are both now unavailable. It is not clear if these actions are related to the week’s events, but both posts were still available until recently.

He went back to Shenzhen where he lives after his call at the summit according to a statement by his spokesman, Ryan Ferrell, and missed a planned look at the summit on November 29th. “I have returned to Shenzhen and will not attend the conference on Thursday. I will stay in China, my home country and fully cooperate with all requests for my work,” said the statement. “

2nd Are his claims correct?

Many researchers have said that an independent body should confirm his scientific statements by making an in-depth comparison between the genes and the children‘s genes. The problem is that most people agree that the children and their parents should remain anonymous. [19659006] “He has kept them secret and for good reasons,” says Nobel’s award-winning biologist David Baltimore, chairman of the Organizing Committee Summit and former president of California Technical University of Pasadena. “We have not even suggested how the independent investigation will happen.”

His team was able to deliver anonymous samples. Outside researchers could also visit his laboratory to analyze the data. In a statement from his spokesman, he said that he will invite other researchers to conduct an independent investigation. “My raw data will be made available for third party review.”

He also says that he has conducted studies on his human rewriting research to magazines for publication. He has told some researchers that a paper will be published by the end of the year, but has not specified any magazine. But even if this happens, strict Chinese genetic resources would prevent him from publishing the gene’s parents or children.

3rd How exactly did CRISPR edit the twins?

In the absence of a peer-reviewed publication or preprint describing his editing work, some researchers analyze their presentation to try to understand how the twin genres were edited – and possible consequences of these changes.

Gaetan Burgio, a geneticist at Australian National University in Canberra, working on CRISPR redirection, said the raw sequencing data he presented in his talk suggest that baby’s cells have multiple edited versions of the CCR5 gene with DNA- deletions of different sizes. Such “mosaicism” can be caused when CRISPR edits some early embryo cells differently than others, or fails to edit any. Other researchers have reported mosaics in attempting to edit human embryos for research purposes.

RNA researcher Sean Ryder at the University of Massachusetts Medical School in Worcester pointed out further concerns in a Twitter post.

] He Jiankui told the redecoration conference that he targeted the CCR5 gene, as some people naturally carry a mutation in CCR5 – a 32-DNA lettering called delta-32 – which inactivates the gene. But Ryder says that the CCR5 erasure he claimed to introduce into the baby’s cells through CRISPR redirection is not identical to the delta-32 mutation. “The point is that none of the three matches the well-studied delta 32 mutation, and as far as I can say, no one has been studied in animal models. Inexplicably,” Ryder wrote in the post.

 Han Jiankui is looking and looking up when answering questions during his presentation on the Human Resource Report on November 28th

He Jiankui claims that he has helped to produce the first children born with edited genomes. Credit: TPG via Zuma

4. When will there be another redundant person?

As Jennifer Doudna, a pioneer in the CRISPR / Cas-9 editorial tool, listened to He presents his work on November 28 this summer, continued an idea to come back to her. “The thought I thought was the possibility for scurcologists to use this unethical. It’s a real risk,” said Doudna, biochemist at the University of California, Berkeley,

. Before revealing, many researchers were already concerned about the prospect of someone was on the brink of creating a refurbished person. Biologist George Daley, Dean at Harvard Medical School in Boston, Massachusetts, and a member of the Summit Organizing Committee pointed to a procedure that replaces diseased mitochondrial DNA into an embryo with fresh mitochondrial DNA from another person, eliminating the embryo’s original pathogenic mutation. Although mitochondrial replacement therapy lacks approval of the biomedical community or the US Food and Drug Administration (FDA), doctors in New York City used to produce a baby in Mexico in 2016. “Similar early practice with embryonic editing of CRISPR / Cas9 is likely despite our requests for caution, “said Daley.

At the Hong Kong Summit, researchers discussed another message on human germline editing – the modification of genes forwarded to future generations – is close. “We are worried about worries,” says Baltimore. “If someone working on the field gets indications In that case it is important that they let the authorities know. “

5. Does he want revelation to prevent ethical efforts to make gender lineage? [19659010] Many researchers fear that he is revelations that could hinder the future of germ plan editing. “In the United States, some draconian prohibitions are proposed, which are anti-science goals,” said Baltimore.

In facts about revelations, FDA Commissioner Scott Gottlieb made comments that raised concerns among researchers. “Governments will now have to react,” he told the biocentury. And on November 28, the American Institute of Health (NIH), Francis Collins, said in a statement that “the need to develop a binding international consensus on setting limits for this kind of research, now under discussion in Hong Kong, has never been more evident . “

The statement released at the summit closes the task of keeping a path open to safely translating the redeployment technique into treatments:” Germline through editing can be acceptable in the future if these risks are handled. “

But the debate has focused on A global interest in germline redirection and fear of a chilling effect can be exaggerated. “There may be some women who are excited about the opportunity to participate in this research,” said Judith Dar, at the University of California Irvine School of Medicine and Law School, at a summit satellite session when asked if the controversy could discourage women from donating eggs for research in the future. “The instinct is to say that this is a debakel and can suppress participation. But I’m always surprised at the different responses,” she added.

6th How will researchers ensure better monitoring of spy editing in the future?

“We have no blueprint but we have asked academies,” said Baltimore. “It is a challenge for the world.”

The statement from the summit organizes the committee proposes that science academies around the world make recommendations to their own governments, while coordinating each other.

It also proposes the creation of an international forum that would conduct research and clinical trials through an international register and discuss issues like fair access to the benefits of redirection. However, reviewing in human embryos has potentially an unpleasant range of users and which can make it difficult to maintain such an organization. “Virtually all laboratories that make molecular biology use this technique,” said Daley.

The committee also proposed the need for a translational on-pathway that would provide a careful and responsible way for researchers to take shortcuts in germline to the clinic. Organization committee member Alta Charo, biologist at the University of Wisconsin-Madison, said the expectations must be realistic. “You can not expect perfection. What you can do is try to minimize these incidents with enforcement that punishes unserious behavior.”

The next human transformation summit will take place in London in 2021.

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