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Phase I Study of Dystrophin Plasmid-Based Gene Therapy in Duchenne/Becker Muscular Dystrophy

✍ Scribed by Romero, Norma B.; Braun, Serge; Benveniste, Olivier; Leturcq, France; Hogrel, Jean-Yves; Morris, Glenn E.; Barois, Annie; Eymard, Bruno; Payan, Christine; Ortega, Véronique; Boch, Anne-Laure; Lejean, Lise; Thioudellet, Christine; Mourot, Brigitte; Escot, Christophe; Choquel, Aurore; Recan, Dominique; Kaplan, Jean-Claude; Dickson, George; Klatzmann, David; Molinier-Frenckel, Valérie; Guillet, Jean-Gérard; Squiban, Patrick; Herson, Serge; Fardeau, Michel


Book ID
120750780
Publisher
Mary Ann Liebert
Year
2004
Tongue
English
Weight
363 KB
Volume
15
Category
Article
ISSN
1043-0342

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## Communicated by Ulf Landegren Duchenne and Becker muscular dystrophy (DMD and BMD) are caused by mutations in the dystrophin gene. Large rearrangements in the gene are found in about two-thirds of DMD patients, with B60% carrying deletions and 5-10% carrying duplications. Most of the remaining