Chinese researcher claims first gene-edited babies

HONG KONG -- A Chinese researcher claims he helped make the world's first genetically edited babies -- twin girls born this month whose DNA he said he altered with a powerful new tool capable of rewriting the very blueprint of life.

If true, it would be a profound leap of science and ethics.

A U.S. scientist said he took part in the work in China, but this kind of gene editing is banned in the United States because the DNA changes can pass to future generations and it risks harming other genes.

Many mainstream scientists think it's too unsafe to try, and some denounced the Chinese report as human experimentation.

The researcher, He Jiankui of Shenzhen, said he altered embryos for seven couples during fertility treatments, with one pregnancy resulting thus far. He said his goal was not to cure or prevent an inherited disease, but to try to bestow a trait few people naturally have -- an ability to resist possible future infection with HIV, the AIDS virus.

He said the parents involved declined to be identified or interviewed, and he would not say where they live or where the work was done.

There is no independent confirmation of He's claim, and it has not been published in a journal, where it would be vetted by other experts. He revealed it Monday in Hong Kong to one of the organizers of an international conference on gene editing set to begin today and earlier in exclusive interviews with The Associated Press.

"I feel a strong responsibility that it's not just to make a first, but also make it an example," He told the AP. "Society will decide what to do next" in terms of allowing or forbidding such science.

Some scientists were astounded to hear of the claim and strongly condemned it.

It's "unconscionable ... an experiment on human beings that is not morally or ethically defensible," said Dr. Kiran Musunuru, a University of Pennsylvania gene editing expert and editor of a genetics journal.

"This is far too premature," said Dr. Eric Topol, who heads the Scripps Research Translational Institute in California. "We're dealing with the operating instructions of a human being. It's a big deal."

However, one famed geneticist, Harvard University's George Church, defended attempting gene editing for HIV, which he called "a major and growing public health threat."

"I think this is justifiable," Church said of that goal.

In recent years scientists have discovered a relatively easy way to edit genes, the strands of DNA governing the body. The tool, called CRISPR-cas9, makes it possible to operate on DNA to supply a needed gene or disable one causing problems.

It's only recently been tried in adults to treat deadly diseases, and the changes are confined to that person. Editing sperm, eggs or embryos is different -- the changes can be inherited. In the U.S., it's not allowed except for lab research. China outlaws human cloning but not specifically gene editing.

He Jiankui, who goes by "JK," studied at Rice and Stanford universities in the U.S. before returning to his homeland to open a lab at Southern University of Science and Technology of China in Shenzhen, where he also has two genetics companies. The university said He's work "seriously violated academic ethics and standards" and planned to investigate. A spokesman for He confirmed he has been on leave from teaching since early this year, but he remains on the faculty and has a lab at the school.

The U.S. scientist who worked with him on this project after He returned to China was physics and bioengineering professor Michael Deem, who was his adviser at Rice in Houston. Deem also holds what he called "a small stake" in -- and is on the scientific advisory boards of -- He's two companies.

The Chinese researcher said he practiced editing mice, monkey and human embryos in the lab for several years and has applied for patents on his methods.

He said he chose embryo gene editing for HIV because these infections are a big problem in China. He sought to disable a gene called CCR5, which forms a protein doorway allowing HIV, the virus causing AIDS, to enter a cell.

All of the men in the project had HIV and all of the women did not, but the gene editing was not aimed at preventing the small risk of transmission, He said. The fathers had their infections deeply suppressed by standard HIV medicines and there are simple ways to keep them from infecting offspring not involving altering genes.

Instead, the appeal was to offer couples affected by HIV a chance to have a child protected from a similar fate.

He recruited couples through a Beijing-based AIDS advocacy group called Baihualin. Its leader, known by the pseudonym "Bai Hua," told the AP it's not uncommon for people with HIV to lose jobs or have trouble getting medical care if their infections are revealed.

Tests suggest one twin had both copies of the intended gene altered and the other twin had just one altered, with no evidence of harm to other genes, He said. People with one copy of the gene can still get HIV, although some very limited research suggests their health might decline more slowly once they do.

Several scientists reviewed materials He provided to the AP and said tests so far are insufficient to say the editing worked or to rule out harm.

They also noted evidence the editing was incomplete and at least one twin appears to be a patchwork of cells with various changes.

"It's almost like not editing at all" if only some of certain cells were altered, because HIV infection can still occur, Church said.

Church and Musunuru questioned the decision to allow one of the embryos to be used in a pregnancy attempt, because the Chinese researchers said they knew in advance both copies of the intended gene had not been altered.

"In that child, there really was almost nothing to be gained in terms of protection against HIV and yet you're exposing that child to all the unknown safety risks," Musunuru said.

The use of the embryo suggests the researchers' "main emphasis was on testing editing rather than avoiding this disease," Church said.