Confirming rumors buzzing for months in scientific circles, Chinese researchers have published a study showing genetic engineering of human embryos for the first time.
The experiments, first reported on Wednesday by Nature News, largely didn't work: Just a few embryos out of dozens were successfully modified. Still, researchers are alarmed because the experiments caused unexpected genetic modifications.
News of the experiments seems likely to restart calls for a temporary halt to this line of research. The new study's leader, Junjiu Huang of China's Sun-yat Sen University, has stopped his lab's work due to the bad results, he told Nature News.
In the study, the researchers manipulated 86 human embryos judged as "non-viable" or incapable of ever leading to a pregnancy. Only 71 embryos survived the DNA modification attempts, which sought to fix a gene responsible for a fatal blood disorder. Of 54 of those surviving embryos, which grew to an eight-cell stage, just seven received the fix. The embryos also were riddled with unexpected, or "off-target," changes to their DNA, the researchers reported.
The study was peer-reviewed in just a day, far quicker than normal, and published in the obscure online science journal Protein & Cell, which is at least partially funded by the Chinese government.
Other researchers, though not surprised by the results, are appalled at their implications.
"The results just underline the level of immaturity of this research," stem cell researcher George Daley of Harvard Medical School told BuzzFeed News. "There is no way this should be used in a clinical setting."
Similar "off-target" effects have been seen in cells treated with the gene modification technique, called CRISPR/Cas9, Daley adds, so the bad results are not too surprising.
But George Church, a Harvard geneticist whose lab pioneered the use of CRISPR, told BuzzFeed News that there are recent improvements in the technology that the Chinese group did not use that could greatly reduce the unwanted effects.
Fears of dangerous attempts to genetically engineer designer babies spurred the call for a research halt last month in the journal Nature and calls by other researchers, including Daley, for broad public discussion of the risks of genetic modification research.
One of the inventors of the CRISPR technology, Jennifer Doudna of the University of California, Berkeley, echoed Daley, saying that the Chinese study underscores that the technology isn't ready to treat disease. Its use, she told BuzzFeed News, "needs to be on hold pending a broader societal discussion of the scientific and ethical issues."
But medical ethicist George Annas of Boston University told BuzzFeed News by email that the Chinese experiments, "demonstrate that a moratorium comes too late and is unenforceable." While scientists may want to do "world first" research, no one should fundamentally alter the human species without wide discussion and agreement, he said. "The fact that the researchers think that they followed ethical guidelines simply speaks to their inability to take the ethics of human embryo research seriously."
Others agree: The new paper is only the beginning. There are rumors of at least one similar study that will be published soon, Paul Knoepfler, a stem cell biologist at the UC Davis School of Medicine in Sacramento, told Buzzfeed News.
"I do worry about stuff like designer babies because it feels like there's this shift towards this use of the technology becoming more of a reality," Knoepfler said. "But then today's paper makes me think that would probably be sort of disastrous."
Other researchers in the U.S. are pushing ahead with what many say is more feasible and less ethically fraught: genetically engineered animals.
Both Daley and Knoepfler suggested that genetic engineering in animal embryos should continue. It "could teach us a lot about the pitfalls and landmines waiting for us in this kind of work," Knoepfler said.
Last year Church and a postdoctoral researcher from his lab, Luhan Yang, started a company called eGenesis that is already working on using the genome editing technology in ways that could benefit humans, but in pigs.
With the development of CRISPR, Church told BuzzFeed News over the phone last month, "the applications were evident right away."
Church and Yang are using CRISPR in pigs in two ways. First, the lab is conducting experiments to humanize certain pig genes so that their organs can be used in human transplants without the risk of infections. The other experiments are looking at ways to engineer pigs that are resistant to several viruses prevalent on pig farms. According to Church, their livestock applications have already garnered interest from some of the biggest pork-producing companies in the world.
"Agricultural applications could be sooner, and if you consider the customer is larger companies and pharmaceutical companies, it could be very, very soon," Church said, estimating that applications could reach the market in the next year or two.
Church says his lab is not currently doing any research with human applications.
Cat Ferguson contributed reporting to this story.
This story has been updated to include comments from Doudna and Annas.
On Wednesday, Francis Collins, director of the National Institutes of Health, announced that the nation's medical research agency will not fund "any use of gene-editing technologies in human embryos." Collins argued that the research has been "almost universally" declared as unethical, unsafe, and of questionable medical value at the present time.
"NIH will continue to support a wide range of innovations in biomedical research, but will do so in a fashion that reflects well-established scientific and ethical principles," Collins wrote.
Dan Vergano is a science reporter for BuzzFeed News and is based in Washington, DC.
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Azeen Ghorayshi is a science reporter for BuzzFeed News and is based in New York. Her PGP Fingerprint is 9739 9DAE 607E A66A 3683 AC20 E34B D2A0 8899 74C4
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