Article ID | Journal | Published Year | Pages | File Type |
---|---|---|---|---|
3079274 | Neuromuscular Disorders | 2013 | 5 Pages |
Abstract
Because it is due to a mutation on the X-chromosome, Duchenne muscular dystrophy rarely affects women, unless there is an unequal lyonisation of the X-chromosome containing the normal dystrophin gene. We report here the unique situation of a symptomatic Duchenne muscular dystrophy woman who was transplanted with myoblasts received from her asymptomatic monozygotic twin sister 20Â years ago. Specific dynamometry was performed to possibly detect a long-term effect of this cell therapy. Long-term safety of myoblast transplantation was established by this exceptional case. However, long-term efficacy could not be definitively asserted for this patient, in spite of several clues suggesting beneficial effects.
Related Topics
Life Sciences
Neuroscience
Developmental Neuroscience
Authors
Jean-Yves Hogrel, Fabien Zagnoli, Aurélie Canal, Bodvael Fraysse, Jean-Pierre Bouchard, Daniel Skuk, Michel Fardeau, Jacques P. Tremblay,