First in a human: gene therapy fixed a one-letter DNA typo inside one patient's liver to treat alpha-1 antitrypsin deficiency, an inherited disease that damages the liver and lungs. The patient now makes the working protein, and no unwanted edits were found. New hope for avoiding a liver transplant.
Coverage of "First in a human: gene therapy fixed a one-letter DNA typo inside one patient's liver to treat alpha-1 antitrypsin deficiency, an inherited disease that damages the liver and lungs. The patient now makes the working protein, and no unwanted edits were found. New hope for avoiding a liver transplant." from 1 source, with a live timeline of who reported what and when.
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