First In-Body Gene Editing Successfully Treats Rare Disease Patient
Published 1 h ago1 sourceEvidence: Reported
A Chinese gene-editing trial for a rare inherited disease in four boys produced mixed outcomes: two improved, one withdrew, and one died from a severe systemic reaction 81 hours after dosing. The death raises safety concerns for the therapy, though the small cohort limits conclusions. The trial's contradictory results underscore the high-risk, high-reward nature of gene-editing treatments.
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