Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders. - Inserm - Institut national de la santé et de la recherche médicale Access content directly
Journal Articles Skeletal Muscle Year : 2011

Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders.

Capucine Trollet
Anne Bigot
Elisa Negroni
Soraya Chaouch
  • Function : Author
  • PersonId : 915880
Annie Wolff
  • Function : Author
  • PersonId : 915881
Prashanth Kandalla
  • Function : Author
  • PersonId : 915882
Solenne Marie
  • Function : Author
  • PersonId : 915883
James Di Santo
  • Function : Author
  • PersonId : 915884
Francesco Muntoni
  • Function : Author
  • PersonId : 915885
Jihee Kim
  • Function : Author
  • PersonId : 915886
Sergiu Blumen
  • Function : Author
  • PersonId : 915888
Thomas Voit
  • Function : Author
  • PersonId : 901909
Woodring Wright
  • Function : Author
  • PersonId : 915889
Ahmed Aamiri
  • Function : Author
  • PersonId : 915890


ABSTRACT: BACKGROUND: Investigations into both the pathophysiology and therapeutic targets in muscle dystrophies have been hampered by the limited proliferative capacity of human myoblasts. Isolation of reliable and stable immortalized cell lines from patient biopsies is a powerful tool for investigating pathological mechanisms, including those associated with muscle aging, and for developing innovative gene-based, cell-based or pharmacological biotherapies. METHODS: Using transduction with both telomerase-expressing and cyclin-dependent kinase 4-expressing vectors, we were able to generate a battery of immortalized human muscle stem-cell lines from patients with various neuromuscular disorders. RESULTS: The immortalized human cell lines from patients with Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, oculopharyngeal muscular dystrophy, congenital muscular dystrophy, and limb-girdle muscular dystrophy type 2B had greatly increased proliferative capacity, and maintained their potential to differentiate both in vitro and in vivo after transplantation into regenerating muscle of immunodeficient mice. CONCLUSIONS: Dystrophic cellular models are required as a supplement to animal models to assess cellular mechanisms, such as signaling defects, or to perform high-throughput screening for therapeutic molecules. These investigations have been conducted for many years on cells derived from animals, and would greatly benefit from having human cell models with prolonged proliferative capacity. Furthermore, the possibility to assess in vivo the regenerative capacity of these cells extends their potential use. The innovative cellular tools derived from several different neuromuscular diseases as described in this report will allow investigation of the pathophysiology of these disorders and assessment of new therapeutic strategies.
Fichier principal
Vignette du fichier
2044-5040-1-34.pdf (1.5 Mo) Télécharger le fichier
2044-5040-1-34.xml (65.56 Ko) Télécharger le fichier
Origin : Publisher files allowed on an open archive
Format : Other

Dates and versions

inserm-00651121 , version 1 (12-12-2011)



Kamel Mamchaoui, Capucine Trollet, Anne Bigot, Elisa Negroni, Soraya Chaouch, et al.. Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders.. Skeletal Muscle, 2011, 1 (1), pp.34. ⟨10.1186/2044-5040-1-34⟩. ⟨inserm-00651121⟩
885 View
522 Download



Gmail Facebook Twitter LinkedIn More