Article

Combining a muscle-tropic AAV capsid with a muscle-specific promoter enhances skeletal muscle gene delivery while reducing off-target expression

2026-06-10

Abstract excerpt

<title>Abstract</title> <p> Objective Viral vector technologies based on adeno-associated viruses (AAV) have demonstrated promising ability to deliver genetic cargo to a range of organs <italic>in vivo</italic> , 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 imp...

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Literature Corpus work: 0e0b99ba-2bc8-5122-8be5-56f191343a05
DOI: 10.21203/rs.3.rs-9793637/v1

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