期刊
YEAR IN HUMAN AND MEDICAL GENETICS: NEW TRENDS IN MENDELIAN GENETICS
卷 1214, 期 -, 页码 199-212出版社
WILEY-BLACKWELL
DOI: 10.1111/j.1749-6632.2010.05836.x
关键词
monogenic diseases; Duchenne muscular dystrophy; forced read-through; exon skipping; personalized medicine
The two therapeutic approaches currently most advanced in clinical trials for Duchenne muscular dystrophy are antisense-mediated exon skipping and forced read-through of premature stop codons. Interestingly, these approaches target the gene product rather than the gene itself. This review will explain the rationale and current state of affairs of these approaches and will then discuss how these gene-derived therapies might also be applicable to other diseases.
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