Overview on applications of antisense-mediated exon skipping

Methods Mol Biol. 2012:867:79-96. doi: 10.1007/978-1-61779-767-5_6.

Abstract

Antisense-mediated exon skipping has multiple therapeutic applications. This chapter gives an overview of how this tool has been employed to restore normal splicing for cryptic splicing mutations, to switch between alternative splicing isoforms, to induce exon inclusion, to correct the reading frame to allow the production of internally deleted proteins, or to induce reading frame disruptions to achieve partial protein knockdown. For each application, examples are discussed and the current state of the art is described.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Exons*
  • Genetic Therapy / methods*
  • Humans
  • Mutation
  • Oligonucleotides, Antisense / therapeutic use*
  • RNA Splicing

Substances

  • Oligonucleotides, Antisense