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


  • eng

Conference Location

  • England