Current Pharmacological Strategies for Duchenne Muscular Dystrophy

Shanshan Yao, Zihao Chen, Yuanyuan Yu, Ning Zhang, Hewen Jiang, Ge Zhang*, Zongkang Zhang*, Baoting Zhang*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

Abstract

Duchenne muscular dystrophy (DMD) is a lethal, X-linked neuromuscular disorder caused by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of muscle stem cells. There is still no cure for DMD so far and the standard of care is principally limited to symptom relief through glucocorticoids treatments. Current therapeutic strategies could be divided into two lines. Dystrophin-targeted therapeutic strategies that aim at restoring the expression and/or function of dystrophin, including gene-based, cell-based and protein replacement therapies. The other line of therapeutic strategies aims to improve muscle function and quality by targeting the downstream pathological changes, including inflammation, fibrosis, and muscle atrophy. This review introduces the important developments in these two lines of strategies, especially those that have entered the clinical phase and/or have great potential for clinical translation. The rationale and efficacy of each agent in pre-clinical or clinical studies are presented. Furthermore, a meta-analysis of gene profiling in DMD patients has been performed to understand the molecular mechanisms of DMD.
Original languageEnglish
Article number689533
JournalFrontiers in Cell and Developmental Biology
Volume9
DOIs
Publication statusPublished - 19 Aug 2021

User-Defined Keywords

  • Duchenne muscular dystrophy
  • pharmacological therapeutics
  • skeletal muscle
  • fibrosis
  • inflammation
  • regeneration
  • meta-analysis

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