CRISPR Successful in New Treatment

June 28, 2021

In a first, physicians have treated patients with an inherited disease by infusing them with the gene-editing tool CRISPR and letting it find its way into defective cells to restore function. This success in treating a rare liver disorder, researchers say, is proof-of-concept that CRISPR can potentially work for some more common diseases. In Issues, Jennifer Doudna, who shared a Nobel Prize for codiscovering CRISPR, sees “vast potential for CRISPR to become a standard of care for treating disease.” But expanding its use, she says, will require “applying it responsibly and allowing it to be fairly assessed by those in need.”

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