Making antisense of splicing.
Journal Article (Journal Article;Review)
Alternative splicing multiplies the coding capacity of the genome, resulting in an expanded proteome that provides many targets for therapy. In addition to creating this diverse pharmacoproteome, the process of splicing can be targeted by conventional and molecular therapies. Splicing as a therapeutic target is highlighted in this review, with a particular emphasis on oligonucleotide-based molecular approaches. These oligonucleotides can be used to promote skipping of constitutive exons, inhibit inappropriately activated exons, or stimulate exons weakened by mutations. Preliminary, but exciting, results suggest that these reagents could have clinical utility in treating previously intractable conditions.
Full Text
Duke Authors
Cited Authors
- Garcia-Blanco, MA
Published Date
- October 2005
Published In
Volume / Issue
- 7 / 5
Start / End Page
- 476 - 482
PubMed ID
- 16248283
International Standard Serial Number (ISSN)
- 1464-8431
Language
- eng
Conference Location
- England