CRISPR corrects mutation in human embryos

First human embryos edited in the USA. Here's why it's problematic.

One of the first steps to this becoming a reality has taken place, with help from a team of worldwide researchers that, for the first time, used CRISPR to correct a mutation that leads to heart conditions in future generations.

They targeted a gene mutation that causes a heart-weakening disease, hypertrophic cardiomyopathy, that affects about 1 in 500 people.

The scientists focused on hypertrophic cardiomyopathy, the most common cause of sudden death in otherwise healthy young athletes.

While researchers laud the breakthrough as a step towards the birth of genetically modified humans and the potential ability to treat inherited genetic diseases, the embryonic humans created and tested in both the United States and Chinese experiments were all destroyed within a few days of the procedure. This is the first use of CRISPR on human embryos in the U.S.

The technique would need to approach 100% effectiveness to be ready for clinical trials aimed at producing pregnancies, Mitalipov said.

"It feels a bit like a "one small step for [hu] mans, one giant leap for [hu] mankind" moment", Jennifer Doudna, a biochemist who helped discover the gene-editing method used, called CRISPR-Cas9, said in an e-mail.

At present, South Korea's ethical guidelines ban experimenting on human embryos in the country.

The researchers started with human embryos created from 12 healthy female donors and sperm from a male volunteer who carried the MYBOC3 gene.

[Recently, ] reports emerged that scientists in OR had used gene-editing technology, known as CRISPR-Cas9, to edit a human embryo. In naturally occurring conceptions, 50 percent of the embryos would have been free of the errant gene.

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. In the remaining 27.6% embryos, the cellular cut-repairing mechanism introduced some unwanted insertions or deletions near the cut.

Previous attempts to extend this technique to human embryos, in China, are reported to have run into problems with mosaicism, where some, but not all, the copies of the relevant gene are changed, so that the embryo develops with different regions expressing different variants of the same gene.

Parents could use the scientific advancement to spare their children from a lifelong debilitating disease. "It's the best way to treat the disease before the genetic mutation is actually transmitted to the embryo".

The research used blastocysts, structures that form in the womb several days after conception and are used in modern in vitro fertilization (IVF) treatments. Any attempt to study germline editing in pregnant women would require permission from the Food and Drug Administration, which is now prohibited by Congress from reviewing any such request. Think of it as a biological cut-and-paste program.

Once the defective elements of the gene had been snipped away, the embryo's own cellular fix systems replaced them with healthy versions. Tests now exist to diagnose many diseases prior to birth; however, at this time, there is no therapy in use that actually alters the DNA of embryos prior to birth.

Earlier this year, an ethics report from the prestigious National Academy of Sciences opened the door to lab research to figure out how to make such changes - but said if germline editing ever is allowed, it should be reserved for serious diseases with no good alternatives and performed under rigorous oversight.

"Although it implies a future in which we could choose to have children without particular genetic diseases, or even design for particular traits, that is still likely a very long time from now", he wrote in an email to KPBS.

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