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First human embryo editing experiment in U.S. corrects gene for heart condition

“Really we didn’t edit anything. Neither did we modify anything,” Mitalipov said. “Our program is toward correcting mutant genes.” Alta Charo, a bioethicist at the University of Wisconsin at Madison who is co-chair of the National Academies committee that looked at gene editing, said that concerns about the work that have been circulating in recent days are overblown. “What this represents is a fascinating, important and rather impressive incremental step toward learning how to edit embryos safely and precisely,” she said. However, “no matter what anybody says, this is not the dawn of the era of the designer baby.” She said that characteristics that some parents might desire, such as intelligence and athleticism, are influenced by multiple genes and that researchers don't understand all the components of how such characteristics are inherited, much less have the ability to redesign them. The research involved eggs from 12 healthy female donors and sperm from a male volunteer who carries the MYBPC3 gene, which causes hypertrophic cardiomyopathy. HCM is a disease that causes an abnormal thickening of the heart muscle but can cause no symptoms and remain undetected until it causes sudden cardiac death. There's no way to prevent Skip Trace or cure it, and it affects 1 in 500 people worldwide. Around the time the sperm was injected into the eggs, researchers snipped out the gene that causes the disease. The result was far more successful than the researchers expected: As the embryo's cells began to divide and multiply, a huge number appeared to be repairing themselves by using the normal, non-mutated copy of the gene from the women's genetic material. In all, they saw that about 72 percent were corrected, a very high number.

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