In 2010, a Rice University graduate student named He Jiankui published a paper describing the nitty-gritty details of a then-arcane bacterial immune system called CRISPR. It was well before scientists unlocked the knowledge that CRISPR could be used to manipulate DNA with a precision and ease that other genome editors lacked.

Now, just eight years later, and after returning to his native China, He has burst onto the global stage in the most spectacular way — an entrance he seems to have meticulously choreographed even as he ignored guidelines established by global scientific panels. Using YouTube rather than an academic journal, He claimed that with the aid of CRISPR, he had helped create the world’s first babies — twin girls born a few weeks ago — whose genomes had been edited as embryos. The announcement dropped like a surprise Beyonce album.

The claim, which has not been verified by outside researchers, was heralded by a few as a scientific milestone, an unprecedented step toward preventing all sorts of diseases. But others viewed it as quite the opposite: the reckless breaking of a scientific taboo for personal gain. After all, many far more experienced and respected researchers had the technical know-how to attempt what He did, but they honored the widely accepted ethical barriers. Such research is also illegal in the United States.

advertisement

He Jiankui (pronounced HEH JEE’-an-qway), who was previously not known to many Western CRISPR experts, immediately found himself at the center of a firestorm of criticism over the lack of transparency in his research, the choice of gene that was edited, and his pursuit of this research at all.

“I am trying to understand what may have motivated the work he describes,” said a scientist who helped organize a major Hong Kong summit on human genome editing that starts Tuesday and who asked not to be named. “As far as I can tell, it was a combination of hubris, naivete, and perhaps a genuine desire to help people in need. He does not seem to have anticipated the profound public backlash against his work and the way it was conducted and publicized.”

advertisement

He clearly knew the attention that his announcement would get. He reportedly worked with an American public relations specialist; gave advance interviews to the Associated Press, which has a global reach; timed the big reveal to the start of the summit; and posted a series of YouTube videos in English celebrating the achievement.

But he also caught the world off guard, keeping his activities hidden from the broader scientific community. Information about his clinical trial, for example, was posted on an online Chinese registry only this month. Experts envisioned the first edited baby to be born after rigorous discussions about regulations and ethics and with strict safeguards in place — all done in a manner that wouldn’t alarm the public.

Even He’s own university, from which he has been on leave since February, distanced itself from the research, saying that it knew nothing about the work and that it was “conducted outside of the campus” and “seriously violated academic ethics and codes of conduct.”

Beyond the condemnation, leading genome editing experts were simply left puzzled by the researcher behind the announcement. He doesn’t have an extensive history of publishing papers about genome editing or embryology or the gene he disabled in the embryos, CCR5, to try to confer resistance to HIV infection. Much of the social media scuttlebutt came back to this question: What was this guy doing editing embryos?

Perhaps He should have been more well-known. In a talk last year at Cold Spring Harbor Laboratory, He discussed editing the genomes of mouse, monkey, and human embryos (editing human embryos in the lab, that is — not for transferring into wombs and starting pregnancies).

That talk earned He a speaking spot at this week’s summit in Hong Kong; his panel is scheduled for Wednesday morning Hong Kong time. In a sign of how just how much He’s CRISPR’d embryos project caught the global scientific community by surprise, however, his speaking slot is not a plenary session, typically reserved for the biggest newsmakers of a conference.

He also co-authored a paper published this week in the CRISPR Journal proposing five “ethical principles” to guide the use of genome editing in the clinic. The second author on the paper: Ryan Ferrell, the American public relations professional who worked with He on the rollout of the CRISPR’d embryos project.

Among the elements He called for: open dialogue.

But He doesn’t seem to have followed his own recommendations.

In April 2016, He wrote to CRISPR pioneer Feng Zhang of the Broad Institute, identifying himself as CEO of his DNA sequencing company Direct Genomics and requesting a tour of Zhang’s lab in Cambridge, Mass. He did not visit then, but he did briefly drop by to visit Zhang’s lab this past August, Zhang told STAT on the sidelines of the genome editing summit.

What did He want to talk about? Methods to reduce off-target effects — cuts CRISPR can make in unintended places — in editing of mouse and human embryos, Zhang recalled.

“It was clear to me that he was having the same challenges as other researchers around lack of efficiency and lack of precision,” Zhang said. “I told him that the technology is neither efficient nor precise enough for real-world application in embryos, including in human IVF applications.”

For Zhang, that conversation has taken on a new tone in light of this week’s news. “Of course, now we know that by August, he must have been quite far along in his work in humans, at least according to his most recent claims,” Zhang said. “He certainly never mentioned this work when I saw him.”

From China to the U.S. and back

He’s career trajectory highlights a few salient facts: His scientific background is wide-ranging, but lacking in deep expertise in CRISPR and embryology. Public records indicate that he is just 34 years old, an age when many top researchers are just opening up their first labs.

He, who appears to have been born and raised in China, earned his undergraduate degree in physics from the University of Science and Technology of China in 2006. He then moved to Texas to earn his Ph.D., in biophysics, at Rice University in Houston; his adviser there, the bioengineering professor Michael Deem, would later collaborate with him on the CRISPR’d embryos project.

After graduating in 2010, He spent about a year doing his postdoc in the lab of Stanford bioengineer Stephen Quake. It was there that He learned about single-molecule sequencing ― a then-revolutionary method of sequencing single DNA molecules without first having to copy them using polymerase chain reaction, according to a 2015 story in the publication Bio-IT World.

He moved back to China around 2012. It’s not clear why he decided to return home, but a few factors may have come into play. He wanted to spin off companies from his academic research back in China, according to Bio-IT World. And financial incentives may have contributed to his decision: He moved back to China as part of the Thousand Talents program, an initiative in which the Chinese government has offered incentives to try to bring back its best and brightest scientists and entrepreneurs who did their training in the U.S.

He opened a lab at the Southern University of Science and Technology of China in Shenzhen. (His current leave from that role is scheduled to last until 2021.). He also started several companies, including Direct Genomics.

He appears to have retained some of the relationships he forged during his training in the U.S. — most notably with Deem, his Rice adviser.

Deem said that the research fit with the work they did on vaccines during He’s time in Houston. In addition to their 2010 study on CRISPR, Deem and He collaborated on studies about the speed of evolution of genes and ways to detect emerging and dangerous strains of influenza.

They were also both listed as authors on a study published last year that described using single-molecule sequencing to unravel the genome of a virus. Other authors on the 2017 paper worked at Direct Genomics.

On Monday, Rice said it had opened an investigation into Deem’s role in the CRISPR’d embryos project, saying the research “violates scientific conduct guidelines and is inconsistent with ethical norms of the scientific community and Rice University.” Deem could not be reached for comment.

Moving fast while preaching caution

In the YouTube videos that He used to unveil his stunning claims to the world, he sits in a nondescript lab with instruments in the background; he wears a light button-up shirt, with no tie and no lab coat. Speaking slowly and in English, He struck a remarkably emotional tone.

He described his work as light years away from creating “designer babies,” which he condemned. He also implored his audience to look beyond the criticism that his work would likely spark, voicing confidence that his project will be viewed favorably by history.

“Please remember that while there may be vocal critics, there are many silent families who have seen a child suffer from genetic disease and should not have to suffer that pain again,” He told the camera in one of the videos. “They may not be the director of an ethics center quoted by the New York Times, but they are no less authorities on what’s right and wrong — because it’s their life on the line.”

Crucially, though, He is not claiming to have tackled genetic disease with his CRISPR’d embryo project. Instead, he claims to have taken on HIV, a disease for which relatively simple ways already exist to keep HIV-positive parents from infecting their children.

And big questions remain about whether He’s project will actually help the family whose babies were purportedly gene-edited. Independent scientists who reviewed some of the documentation said there’s not yet enough evidence to tell whether the editing worked as intended or was safe. They’ve also raised concerns about the implications of He’s admission that one of the gene-edited twins had both copies of CCR5 altered, while the other had only one copy altered.

At the end of another one of He’s YouTube videos, he provides an email address for his lab — and another (DearLuluAndNana@gmail.com) for viewers who want to write to the two gene-edited newborns, who He says are named Lulu and Nana.

While He was slammed for doing too much too fast, he has previously emphasized moving patiently and deliberately when discussing edited embryos.

In his 2017 talk, He raised the 1999 death of Jesse Gelsinger in an early gene therapy trial, which set that field back by more than a decade.

Researchers who might be tempted to use a CRISPR’d embryo to start a pregnancy should remember that case, He suggested.

“I want to remind everyone that we should do this kind of slow and with a bit of caution,” He said, “because a single case of failure may kill the entire field.”

Sharon Begley contributed reporting from Hong Kong.

  • I don’t see much issue with this. We had an IVF baby. We had the opportunity to have various genetic screening done of our embryos. From my point of view, this is just one step earlier in the process of eliminating a potential problem.

  • If true, this man is a hero.

    The fact that he ignored everyone except the parents that wanted the product of his genius and effort is to his credit, despite the hand-wringing that will come from all of the social metaphysicists in the so-called “medical ethics community” (and other such “communities”). Those busy-bodies should have absolutely nothing to say about what is a private transaction between consenting adults. No one else in the entire world is compelled to use or take advantage of this technique, and therefore they should have nothing to say about it. By reason and logic, the decision to use this approach should be entirely up to individuals, so long as no fraud is being committed.

    On the other hand, anyone who is carrying potentially transmissible-through-birth diseases should cheer this man. He appears to have opened the door to massive improvements in the human condition. Indeed, anyone who favors human progress should be ecstatic about this development.

    • On top of it, he didn’t ask for compensation from society as in would have happened here in the US had a couple asked to have this done for them the cost would be a few hundred thousand dollars
      Here the couple wanted it the doctor had the ability they did a private transaction and had he not wanted to spread the word to get more focus on expansion they could have said nothing beyond zero and none would be the wiser
      Time is here that people can choose on their OWN rather then asking the collective approval for everything from F*RTING to breathing if it is OK to do
      Dr. Dave

  • I have to admire him for actually trying to help people rather than sitting contemplating his naval while people get sick around him. Imagine how much good could be done if every researcher was willing to take a risk now and then. Perhaps a few people might suffer as a result, possibly even some die. But if the result is thousands are saved who might otherwise die is society better off? As long as the risks are explained and agreed to willingly by the participants I see no ethical problem in someone willing to take a chance to potentially help others.

  • I don’t think the uproar is that he is not well know, but it is that he did it without peer review or validation which is critical for science. It seems very attention seeking even if well intended. What about all the embryos that it didn’t work for (this was attempted on over 20 embryos and only worked correctly for 1).

  • pedophrasty as defense to ethical and possibly criminal violations… I did it for the children. history will absolve me. rubbish.

    • Looking at history I suggest he is 1000% right when IVF and then cloning first came out everyone went to task over Dolley the sheep and other examples but today it is common and no one thinks twice about calling their OBG and making an appointment for a harvest and IVF session
      I get the ethics side of this but the scientific side someone had to do it so may as well be someone who is ready to rather than playing the Go Set Ready all day long game
      We are so abhorrent of the guys who do it this way rather then Ready Set Go that we have slowed progress down to the least possible that the industry (lead by universities, not commercials) will tolerate or accept. At some point we need to squeeze the trigger and just GO
      Dr. Dave

  • -So he makes a promotional video, then claims he did it for the science? No, he wanted attention.
    -The govt needs to start regulating who can be trained on molecular techniques that could be harmful and wide spreading. CRISPR, MRSA, etc
    -Who will track these two individuals. If one has a right to know if their partner has a disease, then they should have the right know their partner’s genes were edited which will impact their children and decedents indefinitely.

  • It feels like most of the outrage is because he was not a “respected”scientist working in a “prestigious” US institution.
    And in all honesty, editing embryos in a dish is not all that hard if you are a skilled molecular/cellular biologist. The only thing standing between doing it and not doing it is the media backlash and possible consequences for the author’s career/life and ……. maybe, maybe ….. some ethical considerations.

    • TOTALLY true
      This is political, not scientific. Everyone who earns a living by publishing papers or doing research with the intent to publish is up in arms. Anyone who sees the flip side that this could very well resolve many diseases (NOT all and not in every situation) but many simple single gene-based issues can be reduced to old school
      We are working on a single gene disease in dogs and if we are successful we will have given a particular breed of dogs an extended life. We are NOT doing it to publish we are doing it to monetize it for pet owners who want a special breed but don’t want them passing on by age 8 or 9
      Dr. Dave

  • China’s plan is working. They have acquired all of the advanced technical skills they need via their emphasis on placing as many grad students and postdocs in the US and Europe, then repatriating those skills via the Thousand Talents program. Now with their willingness to bypass the ethical blockades, they are in prime position to take the lead in this space (one that may end up being massively lucrative). Whether it ends up being good or bad, impossible to tell. But it’s too late to go back now.

    • Do U think US & Europe would not do the same? They might have done it secretly without anyone knows, that’s how they lead the world in all sorts of technology..

    • Dear faithful patriot,
      without the army of Asian underpaid researchers, in particular from China and other “sh!thole” countries, scientific research in the US would amount to pretty much zero

  • Throw the book at him and lock him up. We need a agency created to stop humam gene manipulation, where hans blick?

    • For what he hasn’t broken any laws! He is working in his OWN company without (presumably) any funding requiring ethics evaluation and in a country with very different requirements than the US and EU
      Although in a bigger scope this could be a mess on the scale and direction he is headed I have to give him kudos for the sack to try it the way he did
      Dr. Dave

Comments are closed.

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy