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 rounds of degeneration and regeneration, muscle atrophy, and myofiber necrosis. There are currently no available cures for DMD and the majority of affected individuals die prematurely from respiratory or cardiac failure. Therefore, there is a great need to develop therapeutic options for DMD patients.
We have recently developed a novel approach named stitchR (stitch RNA). We used stitchR to express a functional human mini-dystrophin gene delivered with a non-integrating viral vector such as AAV as a therapeutic approach to correct DMD. This system has been tested in a mouse model for DMD.
StitchR allows for efficient translation and expression of full-length proteins in mammalian cells. Using StitchR allows for expression of a fully functional Dystrophin gene for both skeletal and cardiac muscle in vivo which can both prevent and reverse disease outcomes in animal models. StitchR can be harnessed for the combination of protein coding functional domains or for the delivery and expression of large protein coding sequences by viral vectors.
Duchenne muscular dystrophy
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