Allele-specific crispr/cas9 correction of a heterozygous dnm2 mutation rescues centronuclear myopathy cell phenotypes

HIGHLIGHTS

  • who: Aymen Rabai from the CentroUniversity, Illkirch, France have published the article: Allele-Specific CRISPR/Cas9 Correction of a Heterozygous DNM2 Mutation Rescues Centronuclear Myopathy Cell Phenotypes, in the Journal: (JOURNAL) of 16/06/2019
  • what: As a paradigm for testing allele-specific genome editing without integration of a selection cassette that might affect the genome structure or regulation, the authors focused on a dominant form of centronuclear myopathy (CNM). The aim of this study was to assess the impact of the most common DNM2 R465W mutation on cellular pathways in myoblasts, providing a cellular . . .

     

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