World's first CRISPR gene-edited babies claimed by Chinese researcher as success

Society will decide what to do next in terms of allowing or forbidding such science: Researcher.

A Chinese researcher claims that he helped make the world’s first genetically edited babies — twin girls born this month whose DNA he claimed to have altered with a powerful new tool capable of rewriting the very blueprint of life.

If true, it would be a profound leap for the sciences as well as ethics.

A US 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.

No proper channel to verify the claim

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 that 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.

He Jiankui during an interview at his laboratory in Shenzhen in China. AP

He Jiankui during an interview at his laboratory in Shenzhen in China. AP

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 that is set to begin Tuesday, and earlier in exclusive interviews with The Associated Press.

Mixed reactions from the scientific community

“I feel a strong responsibility that it’s not just to make a first, but also make it an example,” He told 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.

The researcher's questionable backstory

In recent years scientists have discovered a relatively easy way to edit genes, the strands of DNA that govern the body. The tool, called CRISPR-Cas9, makes it possible to operate on DNA to supply a needed gene or disable one that’s 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, which isn't something the US permits, except in 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 US 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 that 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 US 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.

Part of the IVF procedure in progress. AP

Part of the IVF procedure in progress. AP

The study's intended purpose

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 that forms a protein doorway that allows HIV, the virus that causes 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 that do not involve altering genes.

Instead, the appeal was to offer couples affected by HIV a chance to have a child that might be 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 that it’s not uncommon for people with HIV to lose jobs or have trouble getting medical care if their infections are revealed.

The step-by-step procedure used by the researcher

Here is how He described the work:

The gene editing occurred during IVF, or lab-dish fertilization. First, sperm was “washed” to separate it from semen, the fluid where HIV can lurk. A single sperm was placed into a single egg to create an embryo. Then the gene editing tool was added.

When the embryos were 3 to 5 days old, a few cells were removed and checked for editing. Couples could choose whether to use edited or unedited embryos for pregnancy attempts. 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.

Tests suggest that 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.

The Rice scientist, Deem, said he was present in China when potential participants gave their consent and that he “absolutely” thinks they were able to understand the risks.

Deem said he worked with He on vaccine research at Rice and considers the gene editing similar to a vaccine.

“That might be a layman’s way of describing it,” he said.

Further 'gene-edited' pregnancies on hold 

Both men are physics experts with no experience running human clinical trials.

The Chinese scientist, He, said he personally made the goals clear and told participants that embryo gene editing has never been tried before and carries risks. He said he also would provide insurance coverage for any children conceived through the project and plans medical follow-up until the children are 18 and longer if they agree once they’re adults.

Further pregnancy attempts are on hold until the safety of this one is analyzed and experts in the field weigh in, but participants were not told in advance that they might not have a chance to try what they signed up for once a “first” was achieved, He acknowledged. Free fertility treatment was part of the deal they were offered.

He sought and received approval for his project from Shenzhen Harmonicare Women’s and Children’s Hospital, which is not one of the four hospitals that He said provided embryos for his research or the pregnancy attempts.

Some staff at some of the other hospitals were kept in the dark about the nature of the research, which He and Deem said was done to keep some participants’ HIV infection from being disclosed.

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