enOsCas12f1-mediated exon skipping for Duchenne muscular dystrophy therapy in humanized mouse model

J Genet Genomics. 2024 Feb;51(2):256-259. doi: 10.1016/j.jgg.2023.12.003. Epub 2023 Dec 14.
No abstract available

Publication types

  • Letter

MeSH terms

  • Animals
  • Dystrophin / genetics
  • Exons / genetics
  • Genetic Therapy
  • Mice
  • Muscular Dystrophy, Duchenne* / genetics
  • Muscular Dystrophy, Duchenne* / therapy

Substances

  • Dystrophin