[Duchenne muscular dystrophy: perspectives of treatment].
Although the cloning of the dystrophin gene has led to major advances in the knowledge of the molecular, genetic basis of Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), and other muscular dystrophies, with mutations of genes encoding the dystrophin-associated glycoprotein complex, an effective therapy is still lacking. This review reports some of the most promising pharmacological, molecular and cellular approaches to DMD/BMD. All together, these exciting developments are just puzzling work hypothesis whose clinical developments is on the way.
Palmieri
Beniamino
B
Clinica Chirurgica, Divisione Chirurgia I, Dipartimento Misto di Chirurgia Generale e Specialità Chirurgiche, Università degli Studi di Modena e Reggio Emilia. palmieri@unimo.it
Sblendorio
Valeriana
V
D015316:Genetic Therapy; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy*; D033581:Stem Cell Transplantation