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Gene therapy and antisense methods hold vow to treat Duchenne muscular dystrophy (DMD). The benefits of both healing strategies may be combined by vectorizing antisense sequences into an adeno-associated virus (AAV) vector. We previously reported the effectiveness of AAV-U7 small nuclear RNA (U7snRNA)-mediated exon missing when you look at the mdx mouse, the dys - /utr - mouse, in addition to golden retriever muscular dystrophy (GRM
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