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Articles tagged with adenoassociated virus aav

Adeno-associated virus for the treatment of musle diseases: Toward Clinical trials.

Muscle diseases include musclular dystrophies, cardiomyopathies, neuromuscular and metabolic disorders. The loss of muscular structure and function is associated with significant morbidity and mor...

Tags: Adenoassociated virus AAV, exon skipping.

The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy.

Duchenne muscular dystrophy (DMD) is associated with mutations in the dystrophin gene that disrupt the open reading frame whereas the milder Becker’s form is associated with mutations which ...

Tags: Exon Skipping, adenoassociated virus AAV, mRNA, dystrophin gene.