Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing.
review · Level V
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- Record sourced from PubMed, PMID 12925686.
- Also identified by PMC identifier 171400.
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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.
Medical subject headings
- Alternative Splicing
- Antigens, Surface
- Oligonucleotides, Antisense