Chinese scientist's claim of gene-edited babies 'extremely concerning'

Cristina Cross
November 27, 2018

Prof He alleged that he got rid of the gene CCR5 from the twin girls, Lula and Nana, meaning that they could never contract HIV.

It's not the usual way that reputable scientists announce their breakthroughs to the world, but on Monday, Jiankui He released a video proclaiming that he had produced the world's first human babies whose genomes were edited using the powerful technique called CRISPR.

He had studied in the past at Rice and Stanford universities in the United States.

He Jiankui of Southern University of Science and Technology of China said he altered the DNA of twin girls born earlier this month to try to help them resist possible future infection with the AIDS virus - a dubious goal, ethically and scientifically. If true, many experts say it is a unsafe leap in science and ethics. Mitalipov added. "He's testing his hypothesis on babies". It's like a biological cut-and-paste program: An enzyme that acts like molecular scissors snips a section of a gene, allowing scientists to delete, fix or replace it.

Having one baby born with just one set altered is particularly worrying for reproductive biology expert Dr Hannah Brown, Chief Science Storyteller at the South Australia Health and Medical Research Institute. One first-in-human study is testing intravenous infusion of gene-editing ingredients to fight a killer metabolic disease.

With genomics becoming more widely discussed as a scientific and social topic in recent years, He's gene-edited babies have ignited controversy on Chinese social media. But unlike Monday's announcement, none of those experiments would alter DNA in a way that patients would pass to their own children. That's the story coming out of the country today as researcher He Jiankui is boldly claiming to have performed genetic modification on several embryos from multiple couples, one of which has already resulted in a pregnancy.

In his promotional video, He describes targeting the CCR5 gene, which helps the HIV virus enter healthy human cells. In China, however, only human cloning is outlawed, leaving a gray area when it comes to genetic editing.

Because the research has not yet been published in a scientific journal or carefully vetted by other scientists, many researchers and bioethicists remain cautious about the claim. This technique is banned in the US, because it may cause unpredictable genetic defects in future generations. Plus, long-term negative effects might not become apparent for years.

After He started the work, he gave official notice- on November 8 - on a Chinese registry of clinical trials.

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It's only recently been tried in adults to treat deadly diseases, and the changes are confined to that person.

That lab-only research is going on, by Mitalipov and others.

"Whether the clinical protocols that resulted in the births in China conformed with the guidance in these studies remains to be determined", the group said in a statement. The technology also carries the risk of affecting other genes unintentionally.

No independent outsiders know yet, which is partly why scientists are so disturbed.

Tests of the twins DNA suggest that editing worked on both copies of the gene of one twin and one copy was altered in the other twin.

The CCR5 gene was "turned off", making the babies resistant to HIV, Jianku told the Associated Press.

Nicholas Evans, assistant professor of philosophy at the University of Massachusetts Lowell, said on Twitter that the claims were "wild". In all, 16 of 22 embryos were edited, and 11 embryos were used in six implant attempts before the twin pregnancy was achieved, He said.

The biggest concern: That precision, or lack of it. "For example, he might have made a mutation in a place he didn't intend to make a mutation". In the US, the process is only permitted for lab research. Any pregnancy attempt would require permission from the Food and Drug Administration, which is now prohibited by Congress from even reviewing such a request - a de facto ban.

A physicist by training, He told AP that embryos from seven couples who underwent in vitro fertilisation had been edited. The researchers support studies in which CRISPR is used to develop treatments that would affect cells that aren't passed on to the next generation - i.e. anything except egg and sperm - but say that more research is needed before CRISPR is used to make changes in genomes that can be carried by generation after generation.

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