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Sponsored Artificial Intelligence: A Modern Approach, Global Edition Artificial Intelligence: A Modern Approach, Global Edition

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Jada L. Covington

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Oct 2026

Use of split intein and myotropic AAV vectors enables effective preclinical gene therapy for dysferlinopathy.

Limb-girdle muscular dystrophy type R2 (LGMDR2) is a genetic disorder characterized by progressive skeletal muscle weakness and degeneration. It is caused by mutations in the DYSF gene, which prevent dysferlin expression. Dysferlin protein is essential for muscle cell membrane repair. Gene therapy using adeno-associate...

Nikki M. McCormack, Stephanie Lee-Diaz, Theodore R. Reyes et al. · 0 citations

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