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/ Source: Associated Press
Per Marilynn Marchione, Associated Press
HONG KONG – A Chinese researcher claims he helped create the world's first genetically edited babies – twin girls born this month whose DNA he said altered with a powerful new tool that could rewrite the model of life.
If true, it would be a profound leap in science and ethics.
An American scientist said he took part in the work in China, but this type of genetic editing is banned in the United States because changes in DNA can pass to future generations and can harm other genes.
Many scientists consider it unsafe to try, and some have denounced the Chinese report as a human experimentation.
The researcher, He Jiankui, of Shenzhen, said he changed the embryos to seven couples during fertility treatments, with a resulting pregnancy so far. He said his goal is not to cure or prevent an inherited disease, but to try to give a characteristic that few people have naturally: an ability to resist a possible future infection by HIV, the AIDS virus.
He said that the parents involved did not want to be identified or interviewed, and he did not say where they live or where the work was done.
There is no independent confirmation of He's claim, and it was not published in a journal where it would be examined by other experts. He revealed on Monday in Hong Kong to one of the organizers of an international conference on gene editing that should start on Tuesday and earlier in exclusive interviews with the Associated Press.
"I feel a great responsibility not only to make a first, but also to make it an example," he told the AP. "Society will decide what to do next" in terms of allowing or prohibiting this science.
Some scientists were astonished to hear the statement and condemned it vehemently.
It is "unfair … an experiment on humans that is not morally or ethically defensible," said Dr. Kiran Musunuru, a genetics expert at the University of Pennsylvania and editor of a genetics journal.
"This is very 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, a leading geneticist, George Church of Harvard University, advocated the release of genes for HIV, which he called "a large and growing threat to public health."
"I think that's justifiable," Church said of that goal.
In recent years, scientists have discovered a relatively easy way to edit genes, the DNA strands that govern the body. The tool, called CRISPR-cas9, makes it possible to operate on DNA to provide a needed gene or to disable one that is causing problems.
It has only recently been attempted in adults to treat deadly diseases, and the changes are confined to that person. Sperm, ovule, or embryo editing is different – changes can be inherited. In the US, it is not allowed except for laboratory research. China prohibits human cloning, but not specifically the editing of genes.
He Jiankui, who studies JK, studied at Rice and Stanford universities in the United States before returning to his home country to open a laboratory at the South China University of Science and Technology in Shenzhen, where he also has two genetics companies.
The American scientist who worked with him on this project after he returned to China was professor of physics and bioengineering Michael Deem, who was his adviser at Rice in Houston. Deem also holds what he called "a small turnout" in – and is on the scientific advisory boards of – He is two companies.
The Chinese researcher said that he had been practicing the editing of rats, monkeys and human embryos in the laboratory for several years and applied for patents on his methods.
He said he chose the genetic editing of embryos for HIV because those infections are a big problem in China. He tried to deactivate a gene called CCR5 that forms a protein gateway that allows HIV, the virus that causes AIDS, to enter a cell.
All men in the project had HIV and all women did not, but the genetic editing was not aimed at avoiding the small risk of transmission, he said. Parents have had their infections deeply suppressed by standard HIV medications and there are simple ways to prevent them from infecting offspring that do not involve gene alteration.
Instead, the call was to offer HIV-affected couples a chance to have a child who could 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 is not uncommon for people with HIV to lose jobs or have problems getting medical care if the infections are revealed.
Here's how he described the work:
Genetic editing occurred during in vitro fertilization, or laboratory fertilization. First, the sperm was "washed" to separate it from the semen, the fluid where HIV can hide. A single sperm was placed in a single egg to create an embryo. Then the genetic editing tool was added.
When the embryos were 3 to 5 days old, some cells were removed and checked for editing. Couples may choose to use either published or unissued embryos for attempted pregnancies. In all, 16 of the 22 embryos were edited, and 11 embryos were used in six attempts at implantation 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 only one altered, with no evidence of damage to other genes, he said. People with a copy of the gene may still get HIV, though some very limited research suggests that their health may decline more slowly thereafter.
Several scientists reviewed materials that he provided to the AP and said the tests so far are insufficient to say that the issue worked or to rule out the damage.
They also noted evidence that the issue was incomplete and that at least one of the twins appears to be a patchwork of cells with several changes.
"It's almost like not editing everything," if only some of the cells were changed, because HIV infection could still occur, Church said.
Church and Musunuru questioned the decision to allow one of the embryos to be used in an attempted pregnancy because Chinese researchers said they knew in advance that both copies of the desired gene had not been altered.
"In that child, there was almost nothing to be gained in terms of HIV protection, and yet you're exposing that child to all the unknown security risks," Musunuru said.
The use of this embryo suggests that the "major emphasis of the researchers was on testing the issue rather than avoiding it," said Church.
Even though the edition works perfectly, people without normal CCR5 genes face a higher risk of contracting certain viruses, such as the West Nile, and dying of the flu. As there are many ways to prevent HIV infection and it is very treatable if it occurs, these other medical risks are a concern, Musunuru said.
There are also questions about the way he said he proceeded. He gave official news of his work long after he said it began – on November 8 in a Chinese registry of clinical trials.
It is unclear whether participants fully understood the goal and the possible risks and benefits. For example, consent forms called the project an "AIDS vaccine development" program.
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 Rice's vaccine research and considered vaccine-like gene editing.
"This may be a layman's way of describing this," he said.
Both men are physicists with no experience in clinical trials in humans.
The Chinese scientist, He, said he personally made clear the goals and told participants that the editing of embryo genes has never been tried before and carries risks. He said he would also provide insurance coverage for all children conceived through the project and would plan for medical follow-up until the children are 18 or older if they agree when they are adults.
Other attempts at pregnancy are suspended until the safety of the pregnancy is analyzed and experts in the area analyze it, but participants were not warned in advance that they might not have the chance to try what they signed once a "first" achieved He acknowledged. Free fertility treatment was part of the deal they were offered.
He sought and received approval for his project at the Harmonicare Hospital for Women and Children in Shenzhen, which is not one of the four hospitals he said provided embryos for his research or attempted pregnancies.
Some officials at some of the other hospitals were kept in the dark about the nature of the research, which He and Deem said were made to prevent HIV infection from some participants being reported.
"We think this is ethical," said Lin Zhitong, an administrator at Harmonicare who heads the ethics panel.
Any medical team that dealt with samples that might contain HIV was aware, he said. An embryologist from He's laboratory, Qin Jinzhou, confirmed to the AP that he did sperm washing and injected the gene-editing tool into some pregnancy attempts.
The study participants are not ethicists, he said, but "they are both authorities about what is right and what is wrong because it is their life at risk."
"I believe this will help families and their children," he said. If they cause unwanted side effects or damage, "I would feel the same pain as them and this will be my responsibility."