title: Scalpel or Straitjacket: CRISPR/Cas9 Approaches for Muscular Dystrophies.
abstract: Versatility of CRISPR/Cas9-based platforms makes them promising tools for the correction of diverse genetic/epigenetic disorders. Here we contrast the use of these genome editing tools in two myopathies with very different molecular origins: Duchenne muscular dystrophy, a monogenetic disease, and facioscapulohumeral muscular dystrophy, an epigenetic disorder with unique therapeutic challenges.
authors.affiliation: The Department of Cell and Developmental Biology, University of Massachusetts Medical School, Worcester, MA, 01655, USA.
authors.forename: Charis L
authors.initials: CL
authors.lastname: Himeda
authors.affiliation: The Department of Cell and Developmental Biology, University of Massachusetts Medical School, Worcester, MA, 01655, USA.
authors.forename: Takako I
authors.initials: TI
authors.lastname: Jones
authors.affiliation: The Department of Cell and Developmental Biology, University of Massachusetts Medical School, Worcester, MA, 01655, USA. Electronic address: Peter.Jones@umassmed.edu.
authors.forename: Peter L
authors.initials: PL
authors.lastname: Jones
mesh_terms: D000818:Animals; D064113:CRISPR-Cas Systems; D064112:Clustered Regularly Interspaced Short Palindromic Repeats; D018398:Homeodomain Proteins / Q000235:genetics; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics*
chemical_list: D018398:Homeodomain Proteins
keywords: CRISPR; Cas9; DMD; FSHD; genome editing; muscular dystrophy