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 ...
Skeletal muscle is an attractive site for the delivery and expression of exogenous genes encoding therapeutic proteins for the treatment for systemic diseases and neuromuscular disorders. In this ...
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 ...
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