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Illustration of a DNA double helix National Human Genome Research Institute, public domain

Base editing corrects alpha-1 antitrypsin gene defect in a patient's liver, case report shows

A one-off infusion of an experimental base-editing therapy corrected more than half of the faulty gene copies in a 66-year-old man's liver. The peer-reviewed evidence so far is that single case.

Science Comlumen Desk 3 min read

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    Base editing corrects alpha-1 antitrypsin gene defect in a patient's liver, case report shows

    A one-off infusion of an experimental base-editing therapy corrected more than half of the faulty gene copies in a 66-year-old man's liver. The peer-reviewed evidence so far is that single case.