Molecular genetics of muscle disease--Duchenne and other dystrophies. Future prospects.
In the last few years the biochemical lesion in Duchenne/Becker Muscular Dystrophy has been discovered and the gene for the missing/abnormal protein (dystrophin) has been cloned. The implications of these discoveries on the prospects for diagnosing and eventually eradicating this disease and for the possible treatment of affected boys are discussed.
Lachmann
P J
PJ
D005544:Forecasting; D006801:Humans; D009136:Muscular Dystrophies / Q000235:genetics* / Q000517:prevention & control / Q000628:therapy