AAV-mediated gene transfer of a novel microdystrophin ameliorates pathology and enhances muscle function in a mouse model of DMD.

Of note, the increase in RGX-202-μDys expression in diaphragm did not reach statistical significance at 1 × 10 14 gc/kg, compared to vehicle-treated mdx mice, but was sufficient to suppress fibrosis in the diaphragm ( Figure 4 A; Figure S3 ).

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