title: [Advances in genome editing technologies for treating muscular dystrophy.]
abstract: Recent advances in genome editing technologies have opened the possibility for treating genetic diseases, such as Duchenne muscular dystrophy(DMD), by correcting the causing gene mutations in dystrophin gene. In fact, there are several reports that demonstrated the restoration of the mutated dystrophin gene in DMD patient-derived iPS cell or functional recovery of forelimb grip strength in DMD model mice. For future clinical applications, there are several aspects that need to be taken into consideration:efficient delivery of the genome editing components, risk of off-target mutagenesis and immunogenicity against genome editing enzyme. In this review, we summarize the current status and future prospective of the research in applying genome editing technologies to DMD.
authors.affiliation: Takeda pharmaceutical company Limited, Japan.
authors.forename: Yukimasa
authors.initials: Y
authors.lastname: Makita
authors.affiliation: Center for iPS Cell Research and Application, Kyoto University, Japan.
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Hozumi
authors.forename: Akitsu
authors.initials: A
authors.lastname: Hotta
mesh_terms: D000818:Animals; D004247:DNA / Q000235:genetics; D016189:Dystrophin / Q000235:genetics / Q000378:metabolism; D000072669:Gene Editing; D015316:Genetic Therapy; D016678:Genome; D006801:Humans; D009136:Muscular Dystrophies / Q000235:genetics* / Q000378:metabolism / Q000628:therapy
chemical_list: D016189:Dystrophin; D004247:DNA