Duchenne muscular dystrophy (DMD) is a rapid, progressive disease that usually results in death at around the age of 20, while Becker muscular dystrophy (BMD) is a clinically less severe form of the disease that often has only slight debilitating effects. Deletion mutations in the dystrophin gene have been identified in two thirds of DMD/BMD cases and the clinical progression of DMD or BMD patients can be predicted from whether the deletion disrupts or maintains the translational reading frame of the mRNA [
]. In spite of the progress of molecular understanding of DMD, any effective treatment has not yet been established.
- Monaco A.P.
- Bertelson C.J.
- Liechti-Gallati S.
- Moser H.
- Kunkel L.M.
An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus.
Genomics. 1988; 2: 90-95
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- An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus.Genomics. 1988; 2: 90-95
- Duchenne/Becker muscular dystrophy: from molecular diagnosis to gene therapy.Brain Dev. 1996; 18: 167-172
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Accepted: August 29, 2001
Received: August 9, 2001
© 2001 Elsevier Science B.V. Published by Elsevier Inc. All rights reserved.