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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
Immortalized pathological human myoblasts: Towards a universal tool for the study of neuromuscular disorders
Skeletal Muscle, Volume 1, No. 1, Article 34, Year 2011
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Description
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. © 2011 Mamchaoui et al; licensee BioMed Central Ltd.
Authors & Co-Authors
Mamchaoui, Kamel
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Trollet, Capucine
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Bigot, Anne
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Negroni, Elisa
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Chaouch, Soraya
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Wolff, Annie
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Kandalla, Prashanth K.
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Marie, Solenne
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Di Santo, James P.
France, Paris
Institut Pasteur, Paris
St Guily, Jean L.
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Muntoni, Francesco M.
United Kingdom, London
Ucl Great Ormond Street Institute of Child Health
Kim, Jihee
United Kingdom, London
Ucl Great Ormond Street Institute of Child Health
Philippi, Susanne
France, Paris
Sorbonne Université
Germany, Berlin
Max Delbruck Center for Molecular Medicine
Spuler, Simone
Germany, Berlin
Max Delbruck Center for Molecular Medicine
Lévy, Nicolas
France, Paris
Inserm
Blumen, Sergiu C.
Israel, Hadera
Hillel Yaffe Medical Center
Voit, Thomas
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Wright, Woodring E.
United States, Dallas
Ut Southwestern Medical Center
Aamiri, Ahmed
Morocco, Agadir
Faculté Des Sciences - Agadir
Butler-Browne, Gillian Sandra
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Mouly, Vincent
France, Paris
Sorbonne Université
France, Paris
Centre de Recherche en Myologie
France, Montigny-le-bretonneux
Handicap Neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie Appliquées
Statistics
Citations: 233
Authors: 21
Affiliations: 10
Identifiers
Doi:
10.1186/2044-5040-1-34
Research Areas
Disability
Genetics And Genomics
Health System And Policy