First in-human gene correction fixes one-letter DNA typo in patient's liver
theimposingshadow · reddit · 2026-11-04
In a first for in-human treatment, gene correction therapy repaired a single-letter DNA typo inside a patient's liver to treat alpha-1 antitrypsin deficiency. The patient now produces the working protein, with no unwanted edits detected — new hope for avoiding a liver transplant.
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