Molecular approaches to therapy for Duchenne and limb-girdle muscular dystrophy.
The muscular dystrophies are a heterogeneous group of heritable disorders in which progressive muscle degeneration leads to regional or generalized weakness. Recent advances in molecular genetics, cell biology and vector discovery have improved the outlook for therapeutic intervention. This review focuses on novel approaches to the study of disease pathogenesis and refinements in gene- and cell-based strategies for protein restoration in Duchenne and limb-girdle muscular dystrophy, and concludes with a brief discussion of priorities for future clinical investigation.
Stedman
H H
HH
Department of Surgery and Institute for Human Gene Therapy, University of Pennsylvania School of Medicine, Philadelphia 19104-6160, USA. hstedman@mail.med.upenn.edu
D000818:Animals; D015703:Antigens, CD / Q000235:genetics; D016189:Dystrophin / Q000235:genetics; D022761:Gene Transfer, Horizontal; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D039001:Integrin alpha Chains; D051379:Mice; D009136:Muscular Dystrophies / Q000209:etiology / Q000235:genetics / Q000628:therapy*
D015703:Antigens, CD; D016189:Dystrophin; D039001:Integrin alpha Chains; C085725:integrin alpha7