Article

Efficient Cas9 nuclease-based editing in skeletal muscle via lipid nanoparticle delivery

2025-05-12

Abstract excerpt

Gene editing holds great promise for muscular dystrophy treatment, but the rapid evaluation of different editing modalities in skeletal muscle in vivo remains challenging due to lack of simple, effective delivery tools. Here we demonstrate that selective organ targeting (SORT) lipid nanoparticles (LNP) encapsulating optimized Cas9 cargo can facilitate efficient, local delivery to skeletal muscles achieving editin...

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Literature Corpus work: d3bd2b4f-9cd3-536e-83ce-87ad469162dd
DOI: 10.1101/2025.05.12.653518

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