HONG KONG – A scientist who immediately read the Twitter topic this week, defended his experiment by creating the world's first genetically-mutated, silly and daring way, as he announced, in the press and on YouTube on Wednesday. International Summit on Human Genome Editing.
"In this particular case," he told Jiankai about two girls born as a result of his work, "I feel proud." And far from admitting to any second thought about what colleague scholars slammed as "a terrible act that threatens to set back in the field of therapeutic genomic editing," he doubled: he said that he had begun another pregnancy, at an early stage, with genome-edited embryos.
He began to be more humble in his first public remarks about his research, with a statement coming from the audience: "I have to recover the fact that these results came before the peer review," he said.
In fact, his representatives turned to the reporter months ago, and he allowed extensive filming in his lab to tell a story about his work, as he acknowledged.
He accurately organized a statement on Sunday – two days before the summit – of his allegation that girls born of genome-edited embryos were born a few weeks ago. He was interviewed and collaborated with an extensive film by the Associated Press, who first reported on the news, and posted several online videos in English instead of publishing his research in the academic journal. And when he presented his sliders to the organizers of the summit before the conference, they did not include anything about the work that had already led to the birth.
He also acknowledged that his university was in the dark about what he did.
He commented on a number of prominent scholars. "I do not think this has been a transparent process," said David Baltimore, chair of the California Institute of Technology and Summit, during a long Q & A session after his 20 minute conversation. "We only got to know it after the birth of children. I think that the self-government of the scientific community was not able to do so because of the lack of transparency."
Asked why he was hiding his work from the scientific community, He stressed that he had not done so. He noted that he spoke with Cold Spring Harbor in the USA in 2017 and talked to a scientist at the University of California Berkeley and with the US ethics specialist. But only with the Berkeley scientist, he revealed his plan for the genetic revision of infants.
He also revealed that he has now submitted a research journal, but said he would not fully test the results of the scientific community until this document was published.
Birth cases caused disappointment among scientists here and in the west as it crossed the line agreed by leading world scientific organizations around the world to edit embryonic genomes for reproductive purposes. Such germelin editing causes any change in DNA inheritance for future generations. Genomic edit technology is still relatively new and has technical and security issues, including the risk that DNA will accidentally be cut off in the wrong place.
After his apology for the "leaked", he began to defend his work with silent assurance. People with HIV / AIDS face discrimination, he said, and many children are infected. This led him to select, as his CRISPR target, a gene called CCR5, which, when it is disabled, produces a receptor that is so damaging that HIV can not be used as portal cells. Therefore, people who have mutated CCR5 genes are protected from HIV infection.
His answers were not always convincing, reminding some scholars of the evasiveness of politicians in Sunday morning's talk shows. When Garden Liu from the University of Harvard, the leading developer of genomics editing tools, criticized him for moving forward with an experiment that did not follow the "unmet medical need" (HIV prevention and cure), he said that work was justified because HIV-positive the twin father believed that this technique allowed him to be children who would forever have an immune response to HIV. He also said that he had "personal experience" with HIV, some villages with a rate of infection of 30 percent were known.
"Now it's even more terrible and worse," told Statistics Department Liu, a co-sponsor of the genomic editing company Beam Therapeutics. "His answers show a deeply disturbing naivety about the issues involved. I have deep fears that he might retreat to the field. [of therapeutic genome editing] so bad that patients will not receive the treatment they desperately need. "
Other audience experts were equally critical. "Listening to Dr. He, I can only conclude that it was erroneous, premature, unnecessary and largely useless," said biotechnologist Alta Charo of the University of Wisconsin, member of the High Level Organizing Committee.
Most of His speech described the methods he used and the discoveries he was making in experiments with mice and monkeys, such as the conclusion that genomic editing was most effective when it was completed close to fertilization; He therefore supplied the gene-modifying molecule CRISPR-Cas9 using a microinjection, as well as a semen used to fertilize the egg. But CRISPR "multiple" injections had to increase the effectiveness of editing to ensure that most cells had DNA editing.
He is an expert in DNA sequencing, a method he used to assess whether CRISPR caused unexpected genetic changes. He said that he had found one target for edema in the embryo before implantation, but felt that it would not affect any biological function; when the parents believed that a glitch was acceptable, "the couple was chosen to implant this embryo to begin embryo embryo pregnancy," he said. He added that the sequence made after childbirth did not confirm the editing out of the goal.
Another glue was that only a few early-stage embryonic cells were successfully edited; others retained their original CCR5 gene, which means they can not actually protect against HIV. Again he said that his parents adopted this "mozaicism" and said that he wanted the implantation of the embryo. Finally, with one edit, the CCR5 protein contained five amino acids. It is not clear whether it is serious enough to disable the receptor and thus prevent the infection of HIV, said Liu.
This casual attitude also shook his critics. Harvard Liu, who said that during the conversation he needed to use an antacid tablet to prevent the disease, said it was "absurd" that parents who might have little or no scientific training would make a decision that apparently did not go out help
A large part of the polls focused on His process to obtain informed consent from parents who volunteered to take part in the trial. He acknowledged that he himself had obtained the consent of the couples, not the fact that he had a trained, unincorporated professional.
"I explain that for each family member, after the line and paragraph with the paragraph," the meaning of the informed consent form, "said He, who was criticized for telling patients, the experiment was about the" AIDS vaccine. "In addition, participants agree to protect the" trade secret of the project team " .
"Patients were given a form of consent that falsely stated that it was an AIDS vaccine test and who mixed the study with therapy, claiming they could benefit," Charo told STAT.
He said eight pairs were involved in the study and one was omitted. In each case, his father was HIV positive and his mother was not. He said that a total of 31 embryos were injected and 70 percent were edited.
His response to his work, he answered the question, has led to a clinical study on the use of CRISPR to disable CCR5 human embryos to "stop it because of the current situation." But later he said that the second mother was in a very early stage.
He said the twins, called Lulu and Nana, are healthy and that "there is a plan to monitor children in the next 18 years, hoping that they will agree with adults so that they can continue to monitor and support."
The conversation was unexpectedly due to the fact that he, 34, did not provide any evidence to support the allegations in the AP story. He did not reveal where the twins were born or where their family lived. He told the AP that he had asked for ethical confirmation from the Harmonicare ship at the Women and Children's Hospital in Shenzhen, but that it was not one of four who provided embryos for research leading to birth. The hospital then said that girls were not born there either.
Visitors, several dozen scholars and reporters, had set up a line of 500 people for the glass door in the morning – 7 hours before the start, but there were about 60 blank spaces in the room. Just before His session, three defending workers with prominent hinges stood at positions near the front of the room, as well as the crush of photographers and other journalists who had been inflated, filling the passage and grasping the last available seats (and waiting for the expressions before he spoke before).
"I am well aware that this session is of interest," Robin Lovell-Badge, a biologist at the London-based Francis Crick Institute, said Britain was suspected of having started the session and nearly 5,000 people were watching through television broadcast.
"He needs to be given the opportunity to explain what he has accomplished and why," said Lovell-Bug, adding that "we will immediately close the session" if he speaks of "disobedient behavior".
Unlike other loudspeakers sitting on the front lines and being called on to the stage, when it was time for him to speak, He came from the side door, walking on the pedestal, walking through the applause.
Earlier this week, Harmonicare announced that he had studied any commitments that he had, he studied, and condemned the use of genomics to use IVF embryos used to make pregnancy. The Chinese National Commission for Health has commissioned an official in Guangdong province where he has a Shenzen laboratory to investigate his research, but it is unclear what laws or even guidelines he might have violated in China where the rules on genomic editing and human embryonic research are much freer than many other countries, especially the United States
And the University of Southern University's Chinese Science and Technology University, where he has his own lab, but has been relieved of teaching since the beginning of this year, issued a statement that he did not know about his study, in which he said: "He has seriously violated academic ethics and standards."
A Chinese daily newspaper states that an experiment, for example, He "could be devastating if it was done without strict security guarantees," arguing that such a study "is considered" no. "" He added: "I do not know why he did his experiment. It would be his shame if he did it just to get fame. Therefore, thorough investigation is more than necessary and urgent."
He graduated from the University of Science and Technology in Hefei, Anhui in the eastern province, and then graduated from the USA with Ph.D. 2010 at the University of Rhine, Texas, biophysics. He worked as Stephen Quake of Good Postgraduate Diploma in Stanford University, who returned to China in 2012 to join the Southern University of the last century in the tech-heavy southern city of Shenzhen.
Even as his critique, which continued at the meeting, as well as some formal talks, made sense that the editing of human embryos was inevitable and responsive, as well as a number of recent bioethical reports.
A few hours ago, Dean of Harvard Medical School Dr. George Daley called for the establishment of a "CRISPR embryo editing" standard to assess the competence of the practitioner. "" Privileges granted to individuals [to do this] to be an expert, to be trained, "he said. But he made a courteous eager:" It's time to move on [debates about] ethical admissibility to describe the path to clinical translation … in order to move this technology forward. "
Referring to his manifestly unauthorized and largely unregulated research, Daley said: "The fact that the first human embryo editing event came as a wrong process should not allow us to stick to our neck in the sand … I do not think that one practitioner who resists the rules of the field, is a failure of scientific self-regulation. "
Only a minute later, biologist Kathy Niakan described the experiments that created a big red flag for the use of CRISPR-Cas9 in the embryo. She uses the technique (donated IVF embryos, without the intention of finding pregnancy), disabling some genes, and learning what role they play in early human development.
According to unpublished data, which she described, this method creates what she delicately calls "the complexity of the target". This means that CRISPR-Cas9 has an effect on the gene it was supposed to be, but in addition to its intended editing, it was caused by high levels of DNA deletion and segment loss or growth in the chromosome (in this case 6) containing the target gene.
"This is a remarkably new caution" for editing embryonic genomes, "said Niakan.