Can muscle stem cells from muscular dystrophy sufferers be corrected in a sustained fashion, and ultimately "cured"? Using a gene therapy technique, "exon skipping"1, a Franco-Italian research team coordinated by CNRS researcher Luis Garcia2 and Yvan Torrente3 has achieved this. These researchers have, for the first time, restored the functionality of human dystrophin, the protein that is missing in a setting of Duchenne muscular dystrophy, which is the most common of the neuromuscular diseases. They then transplanted these "corrected" human cells into mouse models of this disease in order to test their efficacy, and the muscle performance of these animals was seen to be improved. Published on December 13, 2007 in the journal Cell
Stem Cell, this work received partial support from the Généthon, the laboratory set up and funded by the AFM thanks to donations to the Téléthon. It constitutes a step forward in autologous cell therapy using the patient's own "rehabilitated" cells.
SOURCE: http://www.institut-myologie.org/anglais/e_upload/pdf/cp_myopathie-anglais.pdf
ON April 9, 2010 at 20:08 hours
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