Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...

Duchenne muscular dystrophy is a genetic disorder caused by mutations in the DMD gene, resulting in a lack of the protein dystrophin. This deficiency leads to progressive muscle weakness and loss of ...

Uses cell’s natural mechanisms for gene repair to induce normal expression of dystrophin Single cut makes minimum genomic modification, at precise location Targets specific cell types to repair: ...

“Our goal is to use a midi-dystrophin or a full-length dystrophin transgene, which contain more functional protein domains than current micro-dystrophins, along with a muscle regulated & targeted AAV ...

Adult skeletal muscle is a tissue with a high capacity for regeneration due to the existence of stem cell population, termed ...

Rescue of dystrophic skeletal muscle in mdx and utrophin/dystrophin-deficient (dko) mouse models by reintroduction of dystrophin has validated gene therapy as a potential therapeutic approach for ...

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Which do you think would win in a fight between gene editing and gene therapy? Well, we might find out sooner than expected, because Vertex Pharmaceuticals (NASDAQ: VRTX) has doubled down on a ...

High-intensity interval training boosts fitness and muscle endurance more effectively than traditional home exercise programs ...

Vertex Pharmaceuticals recently doubled down on its gene editing collaboration with CRISPR Therapeutics, but it's miles behind an arguably simpler solution. DMD is a rare inherited muscle-wasting ...