Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing
- 15 August 2003
- journal article
- Published by American Society for Clinical Investigation in Journal of Clinical Investigation
- Vol. 112 (4) , 481-486
- https://doi.org/10.1172/jci200319547
Abstract
An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that produces splice variants with different functions. Such variants have been linked to a variety of cancers, and genetic diseases such as thalassemia and cystic fibrosis. This Perspective describes a promising approach to RNA repair based on the use of antisense oligonucleotides to modulate alternative splicing and engender the production of therapeutic gene products.Keywords
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