Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy.

A reduction in CK levels was also observed in the 3 × 10 13 vg/kg dose group at 3 months p.i., which did not reach statistical significance (compared with DMD mdx control rats).

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At 6-months p.i., despite the small number of animals in this group and the widespread values, a decreasing trend was seen in the DMD mdx control rats compared with WT control rats ( Figure S9 A).

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(DMD mdx rats ∼8 months old) and at 7–9 months of age (3 months p.i.) in the older rats, the DMD mdx control rats exhibited an increasing trend in LV end-diastolic diameter that was prevented at all vector doses ( Figure 6 A).

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