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In Breakthrough, Scientists Edit a Dangerous Mutation From Genes in Human Embryos 

click to enlarge In an undated handout image, newly fertilized eggs before gene editing. In a major milestone sure to renew ethical concerns, scientists have successfully edited genes in human embryos to repair a common and serious disease-causing mutation, producing apparently healthy embryos, according to a study published in Nature on Aug. 2, 2017. - SHOUKHRAT MITALIPOV VIA THE NEW YORK TIMES
  • Shoukhrat Mitalipov via The New York Times
  • In an undated handout image, newly fertilized eggs before gene editing. In a major milestone sure to renew ethical concerns, scientists have successfully edited genes in human embryos to repair a common and serious disease-causing mutation, producing apparently healthy embryos, according to a study published in Nature on Aug. 2, 2017.

By PAM BELLUCK
© 2017 New York Times News Service

Scientists for the first time have successfully edited genes in human embryos to repair a common and serious disease-causing mutation, producing apparently healthy embryos, according to a study published on Wednesday.

The research marks a major milestone and, while a long way from clinical use, it raises the prospect that gene editing may one day protect babies from a variety of hereditary conditions.

But the achievement is also an example of human genetic engineering, once feared and unthinkable, and is sure to renew ethical concerns that some might try to design babies with certain traits, like greater intelligence or athleticism.
click to enlarge Shoukhrat Mitalipov, director of the Center for Embryonic Cell and Gene Therapy at Oregon Health and Science University, in Beaverton, Ore., March 11, 2014. Mitalipov was a senior author on a study published on Aug. 2, 2017 detailing how scientists have for the first time successfully edited genes in human embryos to repair a serious disease-causing mutation. - LEAH NASH/THE NEW YORK TIMES
  • Leah Nash/The New York Times
  • Shoukhrat Mitalipov, director of the Center for Embryonic Cell and Gene Therapy at Oregon Health and Science University, in Beaverton, Ore., March 11, 2014. Mitalipov was a senior author on a study published on Aug. 2, 2017 detailing how scientists have for the first time successfully edited genes in human embryos to repair a serious disease-causing mutation.

“We’ve always said in the past gene editing shouldn’t be done, mostly because it couldn’t be done safely,” said Richard Hynes, a cancer researcher at the Massachusetts Institute of Technology who co-led the committee. “That’s still true, but now it looks like it’s going to be done safely soon,” he said, adding that the research is “a big breakthrough.”

Scientists at Oregon Health and Science University, with colleagues in California, China and South Korea, reported that they repaired dozens of embryos, fixing a mutation that causes a common heart condition that can lead to sudden death later in life.

If embryos with the repaired mutation were allowed to develop into babies, they would not only be disease-free but also would not transmit the disease to descendants.

The researchers averted two important safety problems: They produced embryos in which all cells — not just some — were mutation-free, and they avoided creating unwanted extra mutations.

Potentially, it could apply to any of more than 10,000 conditions caused by specific inherited mutations. Researchers and experts said those might include breast and ovarian cancer linked to BRCA mutations, as well as diseases like Huntington’s, Tay-Sachs, beta thalassemia, and even sickle cell anemia, cystic fibrosis or some cases of early-onset Alzheimer’s.

“You could certainly help families who have been blighted by a horrible genetic disease,” said Robin Lovell-Badge, a professor of genetics and embryology at the Francis Crick Institute in London, who was not involved in the study.


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