First successful gene-editing of embryos in the United States

August 7, 2017



Fixing mutant genes that can cause heart disease

For the first time in the U.S., doctors have successfully edited a gene in a human embryo using CRISPR technology.

The MYBPC3 gene that was cut from a male donor's DNA is known to cause hypertrophic cardiomyopathy (HCM), a heart disease that affects 1 out of 500 people. HCM has no known cure or treatment as its symptoms don’t manifest until the disease causes sudden death through cardiac arrest.

Human embryos were created from 12 healthy female donors and the sperm from the male volunteer who carried the MYBOC3 gene. As the embryos divided and grew, many repaired themselves using the non-edited genes from the genetic materials of the female donors.

"By using this technique, it’s possible to reduce the burden of this heritable disease on the family and eventually the human population,” - Shoukhrat Mitalipov, Oregon Health and Science University

Gene editing is a controversial area of study, and the researchers’ work included changes to the germ line, meaning the changes could be passed down to future generations.

The embryos were allowed to grow for only a few days and none were implanted into a womb (nor was that ever the researchers’ intention). In fact, current legislation in the U.S. prohibits the implantation of edited embryos. The work conducted by these researchers was well within the guidelines set by the National Academies of Sciences, Engineering, and Medicine on the use of CRISPR to edit human genes.


Original article from Futurism





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