Rogue Gene-Editing Scientist Claims New Pregnancy; Government Probe Launched As Hospital Disavows

The world's first genetically edited babies have been born in China this month, according to Chinese researcher He Jiankui - who says he conducted the research along with US scientist Michael Deem, his advisor at Rice University, who has a "small stake" in He's two Chinese genetics companies.

He has claimed in an AP exclusive report that twin girls born this month had their DNA altered with a powerful new tool which may pave the way for a future free of inherited diseases and undesirable traits, and may even extend one's lifespan and reverse aging. 

What's more, Jiankui says that there is another gene-edited pregnancy in its early stages, according to Wired

Lulu and Nana, the twin girls, aren’t the only children He’s group has Crispr’d. When pressed on the number of implantations that have taken place so far, the scientist disclosed that there is another potential pregnancy involving a gene-edited embryo. He hesitated to answer the question because the pregnancy is in an early stage. His research team has so far injected Crispr systems into 30 embryos that have developed to the blastocyst stage. -Wired

Some have denounced the alleged Chinese report as human experimentation, using methods strictly forbidden in the United States and most other countries. 

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. -AP

He's claim has not been independently confirmed, nor published in a journal where it could be reviewed and vetted by other experts. The claim was made on Monday in Hong Kong to one of the organizers of an international conference on gene editing which kicks off on Tuesday, as well as in an earlier exclusive interview 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 AP, adding "Society will decide what to do next" when it comes to the use or prohibition of 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.” -AP

"If this is a false report, it is scientific misconduct and deeply irresponsible," said professor Robert Winston, Emeritus Professor of Fertility Studies and Professor of Science and Society at Imperial College London. "If true, it is still scientific misconduct," he added. 

Prof Julian Savulescu, an expert in ethics at the University of Oxford, said: "If true, this experiment is monstrous. The embryos were healthy - no known diseases.

"Gene editing itself is experimental and is still associated with off-target mutations, capable of causing genetic problems early and later in life, including the development of cancer.

"This experiment exposes healthy normal children to risks of gene editing for no real necessary benefit." -BBC

CRISPR inventor Feng Zhang, meanwhile, has called for a global moratorium on gene-edited babies

Not everybody thinks He's research was ill-conceived, however; famed Harvard University geneticist, George Church, defended the use of gene editing to combat HIV - which he called a "major and growing public health threat." 

"I think this is justifiable," said Church. 

In response to AP's story, China's health commission said on its website Monday that it has ordered a probe into the research. 

In a filing to the Hong Kong stock exchange after Tuesday's close of trading, meanwhile, the parent company of the Shenzhen hospital linked to the pregnancies, Hormonicare Medical Holdings, said that the facility never participated in any clinincal operation connected to the "gene-edited babies" episode, and that the twins were not delivered in their facility, according to Bloomberg

CRISPR Technology

The science behind He's claimed gene editing is a tool called CRISPR-cas9 - which allows for the substitution or disablement of specific genes. Thus far, the only human experiments have been conducted on adults with deadly diseases, and the changes were confined to that person.

With the editing of sperm and eggs using CRISPR - the changed traits are inherited by future generations

The technique He employed was described as follows: 

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. -AP

He says that mice, monkey and human embryos were experimented on for years before the twins were born, using techniques for which the Chinese scientist has applied for patents. HIV resistance was chosen because the disease is a big problem in China - for which He edited a gene known as CCR5 that allows HIV - the virus which causes AIDS, to infiltrate 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. -AP

According to scientists AP used to verify materials provided by He, the tests so far are insufficient to say that the editing was truly successful, or that no harm would come of it. They also "noted evidence that the editing was incomplete, and that "at least one twin appears to be a patchwork of cells with various changes.

Both Church and Musunuru questioned He's decision to let one of the edited embryos be used in a pregnancy attempt - because it was known in advance that both copies of the intended gene was unaltered. 

"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," said Musunuru. Church, meanwhile, said that the use of that embryo suggests that the "main emphasis was on testing editing rather than avoiding the disease." 

Even if editing worked perfectly, people without normal CCR5 genes face higher risks of getting certain other viruses, such as West Nile, and of dying from the flu. Since there are many ways to prevent HIV infection and it’s very treatable if it occurs, those other medical risks are a concern, Musunuru said.

There also are questions about the way He said he proceeded. He gave official notice of his work long after he said he started it — on Nov. 8, on a Chinese registry of clinical trials.

It’s unclear whether participants fully understood the purpose and potential risks and benefits. For example, consent forms called the project an “AIDS vaccine development” program. -AP

Deem, He's partner, said that he was present in China when the potential participants gave their consent, and says that he "absolutely" thinks that they understood the risks. Both men are notably physics experts, and have no experience running human clinical trials.

In order to conduct the experiments, He sought and received approval from Shenzhen Harmonicare Women's and Children's Hospital - one of four which provided embryos for the experimentation. 

While some staff were unaware of the nature of the experiments in order to keep some participants' HIV status from being disclosed, Harmonicare administrator Lin Zhitong said "We think this is ethical." 

Any medical staff who handled samples that might contain HIV were aware, He said. An embryologist in He’s lab, Qin Jinzhou, confirmed to the AP that he did sperm washing and injected the gene editing tool in some of the pregnancy attempts.

The study participants are not ethicists, He said, but “are as much authorities on what is correct and what is wrong because it’s their life on the line.” -AP

That said, another hospital used - the Southern University of Science and Technology in Shenzhen denied any knowledge of the project and said it will launch an investigation, according to the BBC.  

"I believe this is going to help the families and their children," He said, adding that if it causes unwanted harm or side effects, "I would feel the same pain as they do and it’s going to be my own responsibility."

Where exactly is China headed with all of this?