A Chinese trial of a gene-editing therapy for Duchenne muscular dystrophy produced mixed results, with two of four boys improving and one dying, the South China Morning Post reports.
Results published in the journal Cell on Monday covered HG302, a CRISPR-based treatment from Shanghai-based HuidaGene Therapeutics intended to restore the dystrophin protein. The boys, aged four to eight, each received a single intravenous dose. Two were able to walk further afterwards, one showed no gain and later withdrew, and one died 81 hours after treatment from a severe immune reaction and respiratory failure.
Duchenne is an inherited muscle-wasting disease that affects roughly one in 3,500 to 5,000 boys and often leads to heart and breathing failure by the late 20s or 30s. The therapy has orphan drug status from the US Food and Drug Administration.
Source: South China Morning Post
