[Pharmacological treatments for Duchenne and Becker dystrophies].
Duchenne muscular dystrophy (DMD) is a severe X-linked genetic disease affecting 1 boy out of 3500. DMD is due to the lack of a submembranous cytoskeletal protein named dystrophin, leading to the progressive degeneration of skeletal, cardiac and smooth muscle tissue. A milder form of the disease, Becker muscular dystrophy (BMD), is characterised by the presence of a semi-functional truncated dystrophin, or the full-length dystrophin at reduced level. Three different therapeutic approaches are currently under study, gene therapy, cellular therapy and pharmacological therapy. One of the chosen strategies consists of the overexpression of utrophin, a protein 80% homologous with dystrophin, and able to perform similar functions. In this review, we shall consider studies of pharmacological therapy, the aims of which can be classified in three categories: reversal of dystrophic phenotype, dystrophin expression, utrophin overexpression.
Voisin
Vincent
V
Laboratoire de Neurobiologie Cellulaire et Moléculaire, CNRS UPR 9040, Avenue de la Terrasse, 91198 Gif sur Yvette, France.
de la Porte
Sabine
S
D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D000328:Adult; D000617:Aminoglycosides / Q000627:therapeutic use; D000818:Animals; D002331:Carnitine / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003401:Creatine / Q000627:therapeutic use; D016189:Dystrophin / Q000235:genetics; D015870:Gene Expression / Q000187:drug effects; D015316:Genetic Therapy; D006133:Growth Substances / Q000627:therapeutic use; D006801:Humans; D008297:Male; D051379:Mice; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D055435:Myostatin; D016372:RNA, Antisense / Q000627:therapeutic use; D013654:Taurine / Q000627:therapeutic use; D016212:Transforming Growth Factor beta / Q000627:therapeutic use; D049411:Utrophin / Q000235:genetics
D000305:Adrenal Cortex Hormones; D000617:Aminoglycosides; D016189:Dystrophin; D006133:Growth Substances; C525911:MSTN protein, human; C525912:Mstn protein, mouse; D055435:Myostatin; D016372:RNA, Antisense; D016212:Transforming Growth Factor beta; D049411:Utrophin; D013654:Taurine; D003401:Creatine; D002331:Carnitine