Worries, questions grow on claim of gene-edited babies

Worries, questions grow on claim of gene-edited babies

HONG KONG - AP

In this Oct. 9 photo, Zhou Xiaoqin adjusts a monitor showing a video feed of Qin Jinzhou moving a fine glass pipette containing Cas9 protein and PCSK9 sgRNA to an embryo under a microscope at a laboratory in Shenzhen in southern China's Guangdong province. Chinese scientist He Jiankui claims he helped make world's first genetically edited babies: twin girls whose DNA he said he altered. He revealed it on Nov. 26 in Hong Kong to one of the organizers of an international conference on gene editing.

Leading scientists see even more reasons to worry and have more questions than answers after a Chinese scientist attended an international conference on Nov. 27 and repeated his claim to have helped make the world's first gene-edited babies.

He Jiankui spoke at the gene-editing meeting in Hong Kong where the conference's leader called his experiment "irresponsible" and evidence that the scientific community had failed to regulate itself to prevent premature efforts to alter DNA.

The work is highly controversial because the changes can be inherited and harm other genes. It's banned in some countries including the United States. The scientist, He, says twin girls born earlier this month have DNA altered to make them resistant to possible future infection with the AIDS virus.

"They need this protection since a vaccine is not available," he said to justify his work. Scientists weren't buying it.

"This is a truly unacceptable development," said Jennifer Doudna, a University of California-Berkeley scientist and one of the inventors of the CRISPR gene-editing tool that He said he used. "I'm grateful that he appeared today, but I don't think that we heard answers. We still need to understand the motivation for this."

Doudna is paid by the Howard Hughes Medical Institute, which also supports AP's Health & Science Department.

"I feel more disturbed now," said David Liu of Harvard and MIT's Broad Institute, and inventor of a variation of the gene-editing tool. "It's an appalling example of what not to do about a promising technology that has great potential to benefit society. I hope it never happens again."

There is no independent confirmation of He's claim and he has not yet published in any scientific journal where it would be vetted by experts. At the conference, He failed or refused to answer many questions including who paid for his work, how he ensured that participants understood potential risks and benefits, and why he kept his work secret until after it was done.

After He spoke, David Baltimore, a Nobel laureate from the California Institute of Technology and a leader of the conference, said He's work "would still be considered irresponsible" because it did not meet criteria many scientists agreed on several years ago before gene editing could be considered.

"I personally don't think that it was medically necessary. The choice of the diseases that we heard discussions about earlier today are much more pressing" than trying to prevent HIV infection this way, Baltimore said.

The case shows "there has been a failure of self-regulation by the scientific community" and said the conference committee would meet and issue a statement on Nov. 29 about the future of the field, Baltimore said.

Before He's talk, Dr. George Daley, Harvard Medical School's dean and one of the conference organizers, warned against a backlash to gene editing because of He's experiment. Just because the first case may have been a misstep "should in no way, I think, lead us to stick our heads in the sand and not consider the very, very positive aspects that could come forth by a more responsible pathway," Daley said.

Scientists who go rogue ... it carries a deep, deep cost to the scientific community," Daley said.

Regulators have been swift to condemn the experiment as unethical and unscientific.

The National Health Commission has ordered local officials in Guangdong province to investigate He's actions, and his employer, Southern University of Science and Technology of China, is investigating as well.

On Nov. 27, Qui Renzong of the Chinese Academy of Social Science criticized the decision to let He speak at the conference, saying the claim "should not be on our agenda" until it has been reviewed by independent experts. Whether He violated reproductive medicine laws in China has been unclear; Qui contends that it did, but said, "the problem is, there's no penalty."

He called on the United Nations to convene a meeting to discuss heritable gene editing to promote international agreement on when it might be OK.

Meanwhile, more American scientists said they had contact with He and were aware of or suspected what he was doing.

Dr. Matthew Porteus, a genetics researcher at Stanford University, where He did postdoctoral research, said He told him in February that he intended to try human gene editing. Porteus said he discouraged He and told him "that it was irresponsible, that he could risk the entire field of gene editing by doing this in a cavalier fashion."