Abstract:
:Duchenne and Becker muscular dystrophy (DMD/BMD) are X-linked muscular dystrophies. The isolation of the defective gene in DMD/BMD has led to a better understanding of the disease process and has promoted studies regarding the application of molecular therapy. The purpose of this review is to present the progress made in this area of research with particular reference to dystrophin Kobe. Based on the results from the molecular analysis of dystrophin Kobe, we propose a novel molecular therapeutic method for DMD in which antisense oligonucleotides transform DMD into a milder phenotype by inducing exon skipping. In addition, current proposals for the molecular therapy of DMD are discussed.
journal_name
IUBMB Lifejournal_title
IUBMB lifeauthors
Matsuo Mdoi
10.1080/15216540212333keywords:
subject
Has Abstractpub_date
2002-03-01 00:00:00pages
147-52issue
3eissn
1521-6543issn
1521-6551journal_volume
53pub_type
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