Human DMD gene-editing trial reports limited benefit and fatal immune reaction

Published results from the first human trial of HuidaGene's experimental gene-editing treatment for Duchenne muscular dystrophy show editing of some target genes but limited therapeutic benefit, Nature reported. One of four treated children died after a severe immune reaction. The treatment used an adeno-associated virus to deliver a CRISPR base editor to muscles. Two children received a high dose and two a low dose; the child who died received a high dose. The authors suggest that dosing by body weight might be inappropriate because body weight does not necessarily reflect the amount of target muscle. Safety testing in four monkeys had not detected the fatal reaction seen in the trial.