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Platform based on RNA splicing to produce functional human dystrophy gene for treatment of Duchene Muscular Dystrophy

Background Duchenne muscular dystrophy (DMD) is among the most common fatal genetic diseases in children, occurring in 1 of every ~3,500 live male births. DMD is caused by mutations in the dystrophin gene which is required to maintain muscle fiber integrity. DMD is a debilitating muscle disorder characterized by progressive muscle weakness, cyclic...

Published: 11/21/2022 | Inventor(s): Douglas Anderson

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Category(s): Therapeutic