The story had the ring of a science fiction novel: On Sunday, a Chinese scientist announced the births of twin girls whose genes he claims have been edited in their embryonic stage, ostensibly to reduce their risk to AIDS.
Following the claims by scientist He Jiankui, the backlash from the scientific community was swift, and was echoed Wednesday by Lisette Maddison, the director of Washington State University’s Gene Editing Reagent Core.
“It’s not something that we’re gleefully running towards,” she said. “I think the vast majority of scientists are looking at this and going, ‘Why? Why?’ ”
Gene editing is banned in most countries, including China.
“We’re so not there yet,” Maddison said. “I think that there are a lot of questions that still remain on how effective this technology is going to be as far as impacting the genes that you want to impact and nothing else.”
He’s work was never a question of how. Known as CRISPR, the gene editing technology He allegedly employed is used widely in the scientific community, even in some classrooms. He claims to have used CRISPR to manipulate CCR5, a receptor gene linked to the contraction of AIDS, in the two human embryos.
Maddison’s facility – which launched last year as part of the WSU functional genomics initiative with a focus of bringing gene editing into agriculture – serves the entire WSU system, helping anyone who wants to apply CRISPR gene editing into their scientific work. CRISPR technology allows scientists to edit DNA through two pieces: a protein called Cas9 and guide RNA.
“I call (the guide RNA) the ZIP code director: It recognizes a specific region of DNA, so you can direct the Cas9 protein – which cuts DNA – to a specific point in the genome,” she said.
On Wednesday at the Second International Summit on Human Genome Editing at the University of Hong Kong, He announced that a second woman is pregnant with an embryo edited using CRISPR technology.
During the talk, he said that he had already studied the multigenerational effect of the genetic edit in mice. He said that in the third generation, the study found no differences in wayfinding or exploration behaviors.
However, Robin Lovell-Badge, the forum moderator and a senior group leader at the Francis Crick Institute, pointed out to He that a different study had been done showing that alteration of the CCR5 gene enhanced cognitive abilities. In response to that study, He said he saw the paper and felt that it needed more independent verification.
He also emphasized the need for his work given the severity of the global AIDS problem.
However, many in the scientific community disagree. Drs. Hans-Peter Kiem and Keith Jerome, faculty at the Fred Hutchinson Cancer Research Center, condemned the research claims in a statement Wednesday.
“As scientists studying gene therapies as a potential cure for HIV, we condemn an experiment that reportedly used gene editing technologies on human embryos, blatantly disregarding our international ethical norms,” they wrote.
In a panel discussion following He’s talk, the scientist was asked whether enough is known about CCR5 and its effects on the immune system to choose it as the first gene to manipulate at the embryonic level.
“(I want to start) with something well understood, a single gene, as a conservative first model so that in the future we can move onto multiple gene, more complicated genotypes,” He said at the forum.
Maddison is skeptical as to whether He knows enough about CCR5.
“With the approach that he took, you don’t have a predictable outcome, you just hope you’re going to get something that’s going to lead to something that impacts that gene, in this case in a negative way,” she said. “We have absolutely no idea what the long-term consequence of doing that is. We do know that CCR5 is important for HIV entry, but like most things in biology, a gene doesn’t do just one thing.”
The rationale behind looking at the CCR5 gene is understandable, WSU Clinical Associate Professor Rustin Crutchley said in an email.
“One of the only people ever cured of HIV that we know of is Timothy Brown, a native from Seattle, Washington,” Crutchley said. “He had relapsing acute myeloid leukemia (AML) and received two stem cell transplants from the same donor who did not possess the CCR5 co-receptor. He has not been on antiretroviral treatment for over a decade and has no evidence of HIV infection.”
Still, Crutchley said, He’s study warranted “extreme caution” and echoed Maddison in saying that there were other ways to prevent acquiring HIV.
He said that the parents of the twin girls participated in the work because the father is HIV-infected, but Maddison also questions what steps He took to inform the parents, a process in the medical community known as “informed consent.”
“It’s a huge question on what did the parents know,” Maddison said. “Yes, of course it’s an HIV-infected father, a non-HIV infected mother, so certainly transmission to the baby is a concern, but there are other ways to do this.”
Maddison also said that we can’t be sure whether He edited the embryos at all.
“This is always what’s fraught when ‘science’ gets put out as a press release or YouTube videos before there’s any peer-reviewed information out there,” she said. “Usually when there’s these groundbreaking things, they go through scientific review and people ask that question: Well, prove it to me. In this, everyone is basically taking him at his word that that’s exactly what happened.”
But He said there was inevitability to his actions.
“I feel a strong responsibility not to just make it first but to make it as an example,” He said in a video interview with Associated Press on Monday, when he released his research. “The world will move on to the stage for embryo gene editing. If it’s not me, it’s someone else.”
Associated Press reported on Tuesday that He’s work is under investigation by the Chinese government. He did state at the forum that he was against using gene editing for enhancement.
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