Clinical trials have started for a gene therapy for Duchenne muscular dystrophy, with the first patient having received a dose of the treatment.
Duchenne muscular dystrophy is a progressive genetic condition resulting in severe weakening of the muscles and a shortened lifespan. It is linked to mutations in the gene coding for the protein dystrophin.
The therapy aims to edit the altered version of the gene carried by patients so cells can produce a functional version of dystrophin.
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