gene therapy

A new hope for curing Amyotrophic lateral sclerosis

Dr. Maria Grazia Biferi (Centre of Research in Myology (CRM), Paris) and her team successfully designed, conceived and optimized a gene therapy protocol for ALS (Amyotrophic Lateral Sclerosis).

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

Eric Olson's team used AAV gene therapy vectors to deliver CRISPR gene editing components in a larger animal model of Duchenne Muscular Dystrophy