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New insights in gene‐derived therapy: the example of Duchenne muscular dystrophy
Author(s) -
AartsmaRus Annemieke,
den Dunnen Johan T.,
van Ommen GertJan B
Publication year - 2010
Publication title -
annals of the new york academy of sciences
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.712
H-Index - 248
eISSN - 1749-6632
pISSN - 0077-8923
DOI - 10.1111/j.1749-6632.2010.05836.x
Subject(s) - duchenne muscular dystrophy , genetic enhancement , exon skipping , muscular dystrophy , clinical trial , gene , exon , bioinformatics , medicine , genetics , computational biology , biology , alternative splicing
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.