Challenges in Duchenne muscular dystrophy.
The last seven years has witnessed an explosion in our understanding of the muscular dystrophies. In the early 1980s, prenatal diagnosis of Duchenne muscular dystrophy was developed. The cloning of the gene, in 1996, resulted in a better understanding of the disease process and led to the identification of a novel complex at the membrane. This information led to the cloning of other genes responsible for the autosomally inherited dystrophies. As we approach the millenium, the challenge is shifting to the development of therapy of these diseases. This review, in honour of Professor Alan Emery, explains how these advances have an impact in the clinical management of patients and head the promise the progress holds for the future.
Davies
K E
KE
Department of Biochemistry, University of Oxford, UK. kdavies@bioch.ox.ac.uk
D000818:Animals; D002874:Chromosome Mapping; D003001:Cloning, Molecular; D003598:Cytoskeletal Proteins / Q000235:genetics; D016189:Dystrophin / Q000032:analysis / Q000235:genetics; D015316:Genetic Therapy; D008565:Membrane Proteins / Q000235:genetics; D051379:Mice; D018345:Mice, Knockout; D009136:Muscular Dystrophies / Q000175:diagnosis / Q000235:genetics* / Q000628:therapy; D011296:Prenatal Diagnosis; D049411:Utrophin
D003598:Cytoskeletal Proteins; D016189:Dystrophin; D008565:Membrane Proteins; C483600:Utrn protein, mouse; D049411:Utrophin