Unraveling the role of GDF5 therapeutic potential in Amyotrophic Lateral Sclerosis - Sorbonne Université Access content directly
Conference Poster Year : 2022

Unraveling the role of GDF5 therapeutic potential in Amyotrophic Lateral Sclerosis

T. Marais
  • Function : Author
M. Traoré
  • Function : Author
C. Gentil
  • Function : Author
J. Mésseant
  • Function : Author
B. Cadot
  • Function : Author
S. Cottin
  • Function : Author
M. Zerara
  • Function : Author
L. Strochlic
  • Function : Author
P. Smeriglio
  • Function : Author
M.G. Biferi
  • Function : Author

Abstract

Muscle denervation is a common hallmark of several neuromuscular diseases and accounts for skeletal muscle atrophy and dysfunction associated to their pathophysiology. In non-pathologic muscle, the induction of GDF5/SMAD1/5 pathway is essential for avoiding excessive atrophy but also for promoting re-innervation after nerve damage. Recently, we demonstrated that alterations of GDF5 pathway can be implicated in human and mouse age-related muscle wasting and that its overexpression prevents muscle mass loss and force decline during ageing in mice. SMAD1/5 pathway activation has been described as beneficial for motor neuron dysfunction in an Amyotrophic Lateral Sclerosis (ALS) model. We thus hypothesize that GDF5 implementation could have a positive impact on pathophysiology of the disease. We propose a strategy potentially applicable to different ALS forms and/or to optimize gene therapybased approaches.
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Dates and versions

hal-04002164 , version 1 (02-03-2023)

Identifiers

  • HAL Id : hal-04002164 , version 1

Cite

Sestina Falcone, T. Marais, M. Traoré, C. Gentil, J. Mésseant, et al.. Unraveling the role of GDF5 therapeutic potential in Amyotrophic Lateral Sclerosis. Myology 2022, Sep 2022, Nice (FRANCE), France. ⟨hal-04002164⟩
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