In humanized mouse model of duchenne muscular dystrophy 2institut

HIGHLIGHTS

  • who: Mini-dCas et al. from the State Key Laboratory Medical University, Shanghai, China have published the Article: in humanized mouse model of Duchenne muscular dystrophy 2Institut, in the Journal: (JOURNAL)
  • what: The authors demonstrate a adenine base (mxABE) strategy to treat nonsense mutation-related monogenic diseases via A-to-G in a genetically humanized mouse model of Duchenne muscular dystrophy (DMD).
  • how: To further confirm the causal relationship between c.4174C>T and DMD pathology the authors designed a genetic humanization strategy to generate a personalized mouse model of the effects of . . .

     

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