Gene Therapy Prolongs Lifespan, Alleviates Symptoms of Mice With CLN6 Disease, Study Shows
A one-time treatment with an investigational gene therapy prolongs the lifespan in a mouse model of CLN6 disease, and ameliorates motor, learning, and memory impairments, a study shows. The therapy’s safety and efficacy is currently being evaluated in a Phase 1/2 trial (NCT02725580) in…