CRISPR gene-editing tool has been tried in diseased adults

A Chinese researcher has claimed he helped make the world’s first genetically edited babies — twin girls born this month that represent perhaps the next monumental test of science and ethics.

The researcher, He Jiankui of Shenzhen, said he altered embryos for seven couples during fertility treatments, with one pregnancy so far, the Associated Press reported, citing exclusive access to the researcher, whose discovery was not independently verified.

He said his goal was not to cure or prevent an inherited disease, but to try to bestow a trait that few people naturally have — an ability to resist possible future infection with HIV, the AIDS virus.

In recent years scientists have discovered a relatively easy way to edit genes, known as CRISPR-cas9, which the researcher He used. CRISPR makes it possible to alter DNA to supply a needed gene or disable one that’s causing problems. It’s only recently been tried in adults to treat deadly diseases, and the changes were confined to that person.

A U.S. scientist said he took part in the gene edit work in China, but noted it is banned in the United States because the DNA changes can pass to future generations and it risks harming other genes, the AP said.

He’s claim has not been published in a journal, where it would be vetted by other experts. He did create a promotional video.

He said the motivation for his research was to offer couples affected by HIV a chance to have a child that might be protected from a similar fate.

“I feel a strong responsibility that it’s not just to make a first, but also make it an example,” He told the AP. “Society will decide what to do next.”

The genetic editing of a speck-size human embryo carries significant risks, including the risks of introducing unwanted mutations or yielding a baby whose body is composed of some edited and some unedited cells, said Antonio Regalado, writing for MIT’s Technology Review. Data on the Chinese trial site indicated that one of the fetuses is a “mosaic” of cells that had been edited in different ways, MIT said as part of its own reporting on the discovery.

Some scientists strongly condemned the development.

It’s “unconscionable … an experiment on human beings that is not morally or ethically defensible,” Dr. Kiran Musunuru, a University of Pennsylvania gene editing expert and editor of a genetics journal, told the AP.

The reported discovery surprised the scientific world but is expected to feature in soon-to-begin conference.

“We have never done anything that will change the genes of the human race, and we have never done anything that will have effects that will go on through the generations,” David Baltimore, a biologist and former president of the California Institute of Technology, said in a prerecorded message ahead of the Second International Summit on Human Genome Editing, which begins Tuesday in Hong Kong.

Article via Marketwatch