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Optogenetic modeling of human neuromuscular circuits in Duchenne muscular dystrophy with CRISPR and pharmacological corrections.

Amaia Paredes-RedondoPeter HarleyEleni ManiatiDavid John RyanSandra LouzadaJinhong MengAnna KowalaBeiyuan FuFengtang YangPengtao LiuSilvia MarinoOlivier PourquieFrancesco MuntoniJun WangIvo LieberamYung-Yao Lin
Published in: Science advances (2021)
[Figure: see text].
Keyphrases
  • duchenne muscular dystrophy
  • endothelial cells
  • crispr cas
  • genome editing
  • genome wide
  • induced pluripotent stem cells
  • muscular dystrophy
  • pluripotent stem cells
  • smoking cessation
  • gene expression