Background: Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the DMD gene. These mutations disrupt the dystrophin protein, essential for muscle function. Current treatments, like exon-skipping oligonucleotides, have limitations due to their short lifespan and need for frequent administration.   Objective: This study explored the use of a self-complementary adeno-associated virus (scAAV9) vector expressing U7 small nuclear RNA (snRNA) to target exon 2 of …

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  Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the dystrophin gene, leading to muscle degeneration and early death. Antisense oligonucleotides (AONs) are promising therapeutic agents designed to skip faulty exons in the dystrophin gene and restore functional protein.   This study evaluated the efficiency of AONs targeting exon 53 of the dystrophin gene in a mouse model, using chemically modified AONs to enhance skipping …

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  This study explores the impact of voluntary exercise on the effectiveness of a gene therapy approach for Duchenne muscular dystrophy (DMD) using the D2-mdx murine model. DMD is caused by mutations in the dystrophin gene, leading to muscle damage and impaired regeneration. The research uses adeno-associated virus (AAV)-mediated U7 snRNA to skip a mutation-containing exon and restore dystrophin production. D2-mdx mice were treated with AAV-U7 and allowed to run …

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