HONG KONG –
A Chinese researcher says he helped create the world's first genetically modified babies: two twins born this month whose DNA he said he modified with a powerful new tool capable of rewriting the same plane of life.
If true, it would be a profound leap in science and ethics.
A scientist from the United States said that he participated in work in China, but this type of gene editing is banned in the United States because changes in DNA can be passed on to future generations and risk damaging other genes.
Many conventional scientists think that it is too dangerous to try, and some denounced that the Chinese report is a human experimentation.
The researcher, He Jiankui, of Shenzhen, said he modified the embryos of seven couples during fertility treatments, and so far one pregnancy resulted. He said that his goal was not to cure or prevent a hereditary disease, but to try to give a trait that few people have naturally: the ability to resist a possible future infection with HIV, the AIDS virus.
He said the parents involved refused 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 has not been published in a journal, where other experts would examine it. He revealed on Monday in Hong Kong to one of the organizers of an international conference on gene editing that will begin on Tuesday, and before in exclusive interviews with The Associated Press.
"I feel a great responsibility that it's not just about making a first, but also about being an example," he told the AP. "Society will decide what to do next" in terms of allowing or prohibiting such science.
Some scientists were surprised to hear the claim and strongly condemned it.
It's "inconceivable … an experiment with humans that is not morally or ethically defensible," said Dr. Kiran Musunuru, an expert in genetic editing at the University of Pennsylvania and editor of a genetics journal.
"This is too early," said Dr. Eric Topol, who heads the Scripps Translational Research Institute in California. "We are dealing with the operational instructions of a human being, it's a big problem."
However, a famous geneticist, George Church of Harvard University, defended an attempt at genetic editing for HIV, which he described as "a greater and growing threat to public health."
"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 that govern the body. The tool, called CRISPR-cas9, makes it possible to operate with DNA to supply a necessary gene or disable one that is causing problems.
Only recently has it been tried in adults to treat deadly diseases, and the changes are limited to that person. The edition of sperm, ovules or embryos is different, the changes can be inherited. In the USA UU., It is not allowed, except for laboratory research. China prohibits human cloning but not specifically the editing of genes.
He Jiankui (HEH JEE & # 39; -an-qway), who talks about "JK", studied at Rice and Stanford universities in the USA. UU Before returning to his native country to open a laboratory at the University of Science and Technology of Southern China in Shenzhen, where he also has two genetics companies.
The US scientist UU Who worked with him on this project after his return to China was the professor of physics and bioengineering Michael Deem, who was his adviser at Rice in Houston. Deem also has what he called "a small participation" in, and is part of the scientific advice of, two companies.
The Chinese researcher said he practiced editing mice, monkeys and human embryos in the laboratory for several years and has applied for patents on his methods.
He said he chose the edition of the embryonic gene for HIV because these infections are a big problem in China. He sought to disable a gene called CCR5 that forms a gateway of proteins that allows HIV, the virus that causes AIDS, to enter a cell.
All the men in the project had HIV and all the women did not, but the editing of the gene was not aimed at preventing the small risk of transmission, he said. The parents had their infections deeply suppressed by standard anti-HIV medications and there are simple ways to prevent them from infecting children that do not involve the alteration of the genes.
Instead, the call was to offer couples affected by HIV the opportunity 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 their jobs or have problems getting medical attention if their infections are revealed.
Here is how he described the work:
The edition of the gene occurred during IVF, or fertilization of the laboratory plate. First, the sperm was "washed" to separate it from the semen, the fluid where HIV may be lurking. A single sperm was placed in a single ovule to create an embryo. Then the gene editing tool was added.
When the embryos were 3 to 5 days old, some cells were removed and their editing was verified. Couples can choose whether or not to use edited embryos for pregnancy attempts. In total, 16 of the 22 embryos were published, and 11 embryos were used in six attempts of implants before the twin pregnancy was achieved, He said.
The evidence suggests that one twin had both copies of the desired gene altered and the other twin only one altered, with no evidence of damage to other genes, he said. People with a copy of the gene can still get HIV, although some very limited research suggests that their health may decrease more slowly once they do.
Several scientists reviewed the materials provided to the PA and said that the tests so far are not enough to say that the edition worked or to rule out damage.
They also observed evidence that the edition was incomplete and that at least one twin appears to be a mosaic of cells with several changes.
"It's almost like not editing anything" if only some of the 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 an attempt at pregnancy, because Chinese researchers said they knew in advance that both copies of the desired gene had not been altered.
"In that child, there really was nothing to be gained in terms of protection against HIV and yet he is exposing that child to all unknown security risks," Musunuru said.
The use of that embryo suggests that the researchers' main emphasis was "testing the issue instead of avoiding this disease," Church said.
Even if the editing worked perfectly, people without normal CCR5 genes face greater risks of contracting other viruses, such as West Nile, and dying from the flu. According to Musunuru, there are many ways to prevent HIV infection and it is very treatable if it occurs, those other medical risks are a concern.
There are also questions about the way he said he proceeded. He gave official notice of his work long after he said he started it, on November 8, in a Chinese registry of clinical trials.
It is not clear if the participants fully understood the purpose and possible risks and benefits. For example, the consent forms called the project a "development of AIDS vaccines" program.
Rice's scientist, Deem, said he was present in China when potential participants gave their consent and that he "absolutely" believes they could understand the risks.
Deem said he worked with He on vaccine research at Rice and considers that the gene's edition is similar to a vaccine.
"That could be the way a layman describes it," he said.
Both men are physics experts with no experience in clinical trials in humans.
The Chinese scientist, He, said that he personally made the objectives clear and told the participants that the embryo gene had never been attempted before and involved risks. He said he would also provide insurance coverage for any child conceived through the project and plans a medical follow-up until the children are 18 or older if they agree once they are adults.
Other attempts at pregnancy are pending until the safety of this is analyzed and the experts in the field do, but the participants were not told in advance that they might not have the opportunity to prove what they were enrolled once He achieved the "first", he acknowledged. The free fertility treatment was part of the treatment that was offered to them.
He applied for and received approval of his project at the Women and Children's Hospital of Shenzhen Harmonicare, which is not one of four hospitals that he said provided embryos for his research or attempts at pregnancy.
Some of the staff of some of the other hospitals remained in the dark about the nature of the investigation, which, according to him and Deem, was done to prevent the HIV infection of some participants from being revealed.
"We believe this is ethical," said Lin Zhitong, a manager of Harmonicare who heads the ethics panel.
Any medical staff member who handled samples that might contain HIV was aware, he said. An embryologist in He's lab, Qin Jinzhou, confirmed to the AP that he did the sperm wash and injected the gene-editing tool in some of the pregnancy attempts.
The participants in the study are not ethicists, he said, but "they are so many authorities about what is right as what is wrong because it is their life on the line."
"I think this is going to help families and their children," he said. If it causes unwanted side effects or damage, "I would feel the same pain as them and it will be my responsibility".
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(Copyright © 2018 by The Associated Press, All rights reserved.)