Scientists corrects disease-causing gene mutation in human embryos

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The officials at Oregon Health & Science University said results of the U.S. study would be published in a journal soon.

Shoukhrat Mitalipov and his colleagues from Oregon Health and Science University have successfully used the CRISPR Cas9 gene editing technology to wipe out a genetically inherited heart mutation in embryos.

Despite fears about the new study, "this is not the beginning of designer babies", bioethicist Alta Charo of the University of Wisconsin told BuzzFeed News.

The research was a collaboration between Salk, Oregon Health and Science University and South Korea's Institute for Basic Science.

By using CRISPR to alter the DNA just as the sperm and egg came together, they improved the frequency at which the editing happened, and also how often it was correct.

The team also uncovered a new and potentially important DNA fix mechanism that takes place in early embryo development.


As part of a landmark new study, scientists in the U.S. have successfully edited the DNA of human embryos in an effort to prevent babies being born with genetic mutations and diseases.

Gene editing is still in its infancy so even though this preliminary effort was found to be safe and effective, it is crucial that researchers would continue to proceed with the utmost caution, paying the highest attention to ethical considerations. 72% of the embryos (42 of 58) were adjusted so that the rate would have been 50% without the famous scissors genetic (naturally, the embryos would have had a chance on two to inherit a healthy gene).

Other scientists hailed the development as a major breakthrough that could help tackle some of the worst heriditary diseases - while calling for discussion and a tight regulatory handle on the situation every step of the way. Having one bad copy of this gene can lead to heart failure and sudden death. The process works in three steps. Then, the cut is spontaneously repaired by the cell with different mechanisms: one repairs the DNA without leaving any trace, while the other introduces some unwanted insertions or deletions of a few base pairs near the cutting site. Such mosaic embryos probably arise when the fertilized egg copies its DNA before researchers add Cas9, Mitalipov says. And CRISPR didn't target any other genes - collateral damage that plagued similar Chinese studies in 2015. As for the possibility of "designer babies", CRISPR technology isn't almost advanced enough to make anything approaching that feasible, as Belmonte explains.

In December 2015, an worldwide group of scientists convened by the us national Academy of sciences (NAS) in Washington had estimated that it would be "irresponsible" to use the technology of CRISPR to modify the embryo for therapeutic purposes as long as issues of safety and effectiveness have not been resolved. Wu stressed that the new study is squarely in the "basic research" category. The research protocols banned implantation in the womb.

"Unfortunately, we live in a world in which scientists, if they don't like the laws in one country, can go to another country", he says.

Early days as it is, the new study brings up interesting ethical questions. First a segment of RNA is coded with instructions to locate a specific part of the genome by copying that genetic sequence. "So there is still a long road ahead, particularly if you want to do it in a regulatory way". There are an very bad lot of unknowns. "In anticipation of that use, it is essential that the general public, not just the scientists and clinicians, needs to learn about and consider the implications of the potential applications". But there's wide agreement that more research is needed before ever testing the technique in pregnancy.

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