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AAVGene TherapyMuscular Dystrophy
January 27, 2026 Jesse Damsker Comments: 0

Toward an AAV Gene Therapy to Treat Muscular Dystrophies

New innovations in AAV vector engineering — including dual-vector methods and enhanced tissue targeting — are opening doors for treating muscular dystrophies once considered untreatable....
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AAVAlternative SplicingCardiac InvolvementCHKBCHKB MutationsClinical StrategiesDuchenne Muscular DystrophyFunctional GenomicsGene DeliveryGene EditingGene TherapyGenetic TherapeuticsMDCMCMuscular DystrophySafetyTherapeutic StrategyViral VectorsWhole Exome Sequencing
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