Peter Sazani, Ryszard Kole
J Clin Invest.
2003;
112(4):481–486
doi:10.1172/JCI19547
This article Copyright © 2003, The American Society for Clinical Investigation
Abstract
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n 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.
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