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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
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Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy

Published on: January 12, 2019

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Targeting RAGE as a potential therapeutic approach to Duchenne muscular dystrophy

Roberta Sagheddu1,2, Sara Chiappalupi1,2, Laura Salvadori1,2

  • 1Department of Experimental Medicine, University of Perugia, Perugia, Italy.

Human Molecular Genetics
|August 8, 2018
PubMed

Insights

Blocking Receptor for Advanced Glycation End-products (RAGE) reduces inflammation and muscle damage in Duchenne muscular dystrophy (DMD) models. This suggests RAGE inhibition as a potential therapy for DMD patients.

Area of Science:

  • Muscle Biology
  • Immunology
  • Genetics

Background:

  • Duchenne muscular dystrophy (DMD) is a severe X-linked muscle-wasting disease characterized by progressive degeneration and chronic inflammation.
  • Receptor for Advanced Glycation End-products (RAGE) is implicated in inflammation and muscle repair, re-expressed during muscle injury and in certain myopathies.

Purpose of the Study:

  • To investigate the role of RAGE in the pathology of DMD using the mdx mouse model.
  • To evaluate the therapeutic potential of targeting RAGE in DMD.

Main Methods:

  • Generated and analyzed mdx/Ager-/- double mutant mice lacking dystrophin and RAGE.
  • Assessed muscle inflammation, fibrosis, strength, and macrophage responsiveness in mdx and mdx/Ager-/- mice.
  • Administered RAGE-blocking antibodies in vivo to mdx mice.

Main Results:

  • mdx/Ager-/- mice exhibited reduced muscle inflammation, preserved muscle strength, and unaffected fibrosis compared to mdx mice.
  • Macrophages from mdx/Ager-/- mice showed decreased responsiveness to pro-inflammatory stimuli and lower expression of chemotactic factors.
  • In vivo RAGE blockade in mdx mice reduced muscle necrosis and inflammatory infiltrate.

Conclusions:

  • RAGE signaling is chronically activated in DMD muscles and contributes to sustained inflammation and necrosis.
  • Inhibiting RAGE activity may offer a therapeutic strategy to mitigate muscle inflammation and improve muscle function in DMD.

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