The SINE Compound KPT-350 Blocks Dystrophic Pathologies in DMD Zebrafish and Mice

Academic Article

Abstract

  • Hightower et al. demonstrate the therapeutic use of a SINE (selective inhibitor of nuclear export) compound, KPT-350, to treat Duchenne muscular dystrophy (DMD) pathologies in two vertebrate animal models. The authors demonstrate these effects are due to an increase in a pro-regenerative macrophage population in dystrophic skeletal muscles. KPT-350 is in clinical trials for other neuromuscular disorders and may hold promise to ameliorate DMD symptoms.
  • Published In

  • Molecular Therapy  Journal
  • Digital Object Identifier (doi)

    Author List

  • Hightower RM; Reid AL; Gibbs DE; Wang Y; Widrick JJ; Kunkel LM; Kastenschmidt JM; Villalta SA; van Groen T; Chang H