Chinese scientist says first gene-edited babies have been born in effort to fight HIV

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He Jiankui
Chinese researcher He Jiankui discusses his lab’s effort to produce babies whose genes have been altered to protect them from future HIV infection. (The He Lab via YouTube)

A Chinese researcher says his lab facilitated the first birth of gene-edited children — twin girls who are said to possess genetic alterations that could protect them from HIV, the virus that causes AIDS.

“Two beautiful little Chinese girls, named Lulu and Nana, came crying into this world as healthy as any other babies a few weeks ago. The girls are home now,” He Jiankui, a researcher at the Southern University of Science and Technology in Shenzhen, said in a YouTube video.

If confirmed, the report is certain to bring the ethical issues surrounding human genetic engineering into sharp focus, and could lead either to rapid developments in the technology or regulatory limits.

He’s lab made clear that Lulu and Nana were pseudonyms, used to protect privacy. The names that He gave for the parents, Mark and Grace, are presumably pseudonyms as well.

In a series of videos, He said his lab devised the experiment to help parents with the HIV virus feel more confident about having children. Mark, the father of Lulu and Nana, has HIV, the researcher said.

“Employers fire people like Mark,” He said. “Doctors deny medical care, and even forcibly sterilize women. Mark and Grace couldn’t bear to bring a child into that world of fear. … A gene surgery that could save a child from a lethal genetic disease like cystic fibrosis or life-threatening infection like HIV doesn’t just give that little boy or girl an equal chance at a healthy life. We heal a whole family.”

The Associated Press quoted He as saying that he practiced using the CRISPR gene-editing technique on animal subjects for several years. Then, working through an advocacy group for HIV patients, He’s lab signed up couples for in-vitro fertilization. According to the AP account, researchers altered 16 embryos to disable a gene known as CCR5, which plays a key role in the spread of the HIV virus.

“This surgery removed the doorway through which HIV enters to infect people,” He said on video.

Genetic tests conducted after the girls’ birth confirmed that changes in the CCR5 gene persisted, the researcher said.

AP quoted He as saying that both copies of the CCR5 gene had been disabled for one of the girls, while only one gene of the pair was disabled for the other girl. At least one of the twins appeared to have a patchwork of altered and unaltered cells, scientists familiar with the experiment told AP.

There was no independent confirmation of the experiment’s results, which have not yet been published in a peer-reviewed journal. The reports about the Chinese clinical trial coincide with an international summit on gene editing that’s due to begin in Hong Kong later this week.

AP’s report suggests that outside researchers are already raising questions about the ethics of the experiment. Harvard geneticist George Church, a pioneer in the field of gene editing, was quoted as saying that He’s goal was “justifiable” but questioned whether the partially altered embryo should have been implanted.

There are also questions about how much information about the experiment was given in advance to Chinese health officials or to the parents. Moreover, the genetic mutation in the CCR5 gene that protects against HIV is known to raise a person’s vulnerability to flu viruses and the West Nile virus.

The experiment’s lead researcher said he was ready to take the heat.

“I understand my work will be controversial,” He said, “but I believe families need this technology, and I’m willing to take the criticism for them.”

The controversy is sure to continue in the weeks and months ahead. And the first task will be to get outside confirmation that He and his team have actually done what they say they’ve done. In this regard, the scandal that erupted more than a decade ago over fraudulent claims of human embryonic stem cell cloning in South Korea serves as a cautionary tale.




Source: GeekWire



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