Journal
CELLULAR AND MOLECULAR LIFE SCIENCES
Volume 60, Issue 5, Pages 825-833Publisher
SPRINGER BASEL AG
DOI: 10.1007/s00018-003-3042-7
Keywords
antisense; splicing; oligonucleotides; snRNAs; gene therapy
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Antisense technology has been used to study basic biological processes, and to block these processes when they deleteriously lead to human disease. A separate, equally important application of antisense technology is to upregulate the gene expression lost in the diseased state by shifting alternative splicing of pre-messenger RNA. This strategy has commonly relied upon the use of antisense oligonucleotides; however, another approach is to use a plasmid construct to generate antisense RNA inside the cell. Antisense therapeutics based on expression vectors and viral vectors offers a gene therapy approach, whereas those based on oligonucleotides offers a more drug like approach.
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