Cellular Therapies for Muscular Dystrophies: Frustrations and Clinical Successes

Hum Gene Ther. 2016 Feb;27(2):117-26. doi: 10.1089/hum.2015.139. Epub 2016 Jan 25.

Abstract

Cell-based therapy for muscular dystrophies was initiated in humans after promising results obtained in murine models. Early trials failed to show substantial clinical benefit, sending researchers back to the bench, which led to the discovery of many hurdles as well as many new venues to optimize this therapeutic strategy. In this review we summarize progress in preclinical cell therapy approaches, with a special emphasis on human cells potentially attractive for human clinical trials. Future perspectives for cell therapy in skeletal muscle are discussed, including the perspective of combined therapeutic approaches.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cell- and Tissue-Based Therapy / methods*
  • Clinical Trials as Topic
  • Humans
  • Mice
  • Muscle Development
  • Muscle, Skeletal / pathology
  • Muscular Dystrophies / pathology
  • Muscular Dystrophies / therapy*
  • Myoblasts / cytology
  • Myoblasts / physiology
  • Myoblasts / transplantation*
  • Pericytes / cytology
  • Pericytes / physiology
  • Pericytes / transplantation*
  • Pluripotent Stem Cells / cytology
  • Pluripotent Stem Cells / physiology
  • Pluripotent Stem Cells / transplantation*
  • Satellite Cells, Skeletal Muscle / cytology
  • Satellite Cells, Skeletal Muscle / physiology
  • Satellite Cells, Skeletal Muscle / transplantation*
  • Transplantation, Autologous
  • Transplantation, Homologous
  • Treatment Failure