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Here we demonstrate that selective organ targeting (SORT) lipid nanoparticles (LNP) encapsulating optimized Cas9 cargo can facilitate efficient, local delivery to skeletal muscles achieving editing rates \u226535% and restore protein expression for a proof-of-concept muscular dystrophy target. Interestingly, efficient editing in skeletal muscle was observed despite a strong adaptive immune response to repeat dosing of the Cas9 LNPs. High efficiency editing mediated by LNP-based delivery of Cas9 to skeletal muscle permitted detailed analysis of insertion and deletion (InDel) outcomes\n                  <jats:italic>in vivo<\/jats:italic>\n                  for a set of potential therapeutic target sites, which differed substantially from InDel outcomes observed in proliferating cells in one specific instance. Overall, our findings on enhanced LNP delivery of Cas9, platform-specific immune responses, and differential editing patterns observed between\n                  <jats:italic>in vitro<\/jats:italic>\n                  and\n                  <jats:italic>in vivo<\/jats:italic>\n                  models provide valuable insights that should inform the development of gene editing therapeutics for neuromuscular diseases.\n                <\/jats:p>\n                <jats:sec>\n                  <jats:title>One Sentence Summary<\/jats:title>\n                  <jats:p>SORT LNPs permitted efficient Cas9-mediated repair of a pathogenic allele in skeletal muscle in a mouse model of LGMDR7.<\/jats:p>\n                <\/jats:sec>","DOI":"10.1101\/2025.05.12.653518","type":"posted-content","created":{"date-parts":[[2025,5,13]],"date-time":"2025-05-13T01:05:17Z","timestamp":1747098317000},"source":"Crossref","is-referenced-by-count":3,"title":["Efficient Cas9 nuclease-based editing in skeletal muscle via lipid nanoparticle delivery"],"prefix":"10.64898","author":[{"given":"Sukanya","family":"Iyer","sequence":"first","affiliation":[{"name":"Department of Molecular, Cell and Cancer Biology, University of Massachusetts Chan Medical School; 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