Systemic gene delivery into muscle has been a major challenge for muscular dystrophy gene therapy, with capillary blood vessels posing the principle barrier and limiting vector dissemination. Previous ...

In utero gene delivery could offer the advantage of treatment at an early stage for genetic disorders such as Duchenne muscular dystrophy (DMD) in which the inevitable process of muscle degeneration ...

Non-human primate study results demonstrate improvements in skeletal and cardiac muscle delivery efficacy, safety potential, human translatability, and manufacturability over prior AAV capsids ...

PARIS, France (September 12, 2024) - Genethon, the pioneering French laboratory and leader in the research and development of gene therapies for rare diseases, announced today publication in Nature ...