Article

Refining gene delivery to skeletal muscle with a dual-strategy approach of muscle-tropic AAV capsids and muscle-specific promoters

2024-08-06

Abstract excerpt

Viral vector technologies based on adeno-associated virus (AAV) have demonstrated promising ability to deliver genetic cargo to a range of organs in vivo, with several novel candidates showing clinical efficacy in human trials over the past decade. However, naturally occurring AAV serotypes are limited in their ability to target skeletal muscle, an important gene therapy target for many neuromuscular disorders. T...

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Literature Corpus work: a089f30a-4595-5743-98b9-8a88ae1edfdc
DOI: 10.1101/2024.08.02.605568

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