Article

Novel Muscle-Tropic AAV Capsids with Dramatically Enhanced Transduction and Safety Profiles in Non-Human Primates

2026-05-26

Abstract excerpt

<h4>ABSTRACT</h4> Adeno-associated virus (AAV) gene therapy holds immense promise for treating muscular dystrophies, yet its efficacy and safety are constrained by the suboptimal tissue tropism of natural serotypes. Here, we employed the REACH platform, which combines rational design and directed evolution, to engineer muscle targeting vectors. Systemic administration in non-human primates (NHPs) revealed that le...

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Literature Corpus work: da01c947-fe82-5edb-b3be-05be358ea217
DOI: 10.64898/2026.05.22.727076

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