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Genetic correction strategies for Duchenne Muscular Dystrophy and their impact on the heart.

Jamie R JohnstonElizabeth M McNally
Published in: Progress in pediatric cardiology (2021)
Many genetic treatment strategies are being implemented to treat DMD. Since most preclinical testing has focused on skeletal muscle, there is a gap in knowledge about the expected effects of these approaches on cardiac genetic correction and cardiomyopathy progression in DMD. Additional study is needed.
Keyphrases
  • duchenne muscular dystrophy
  • skeletal muscle
  • genome wide
  • heart failure
  • muscular dystrophy
  • copy number
  • healthcare
  • left ventricular
  • insulin resistance
  • stem cells
  • adipose tissue
  • mesenchymal stem cells