Making antisense of splicing

Research output: Contribution to journalReview article

19 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)476-482
Number of pages7
JournalCurrent Opinion in Molecular Therapeutics
Volume7
Issue number5
StatePublished - Oct 1 2005
Externally publishedYes

Keywords

  • Alternative splicing
  • Dystrophin
  • Oligonucleotides
  • Pre-mRNAs
  • Splice sites
  • Splicing

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics
  • Pharmacology
  • Drug Discovery
  • Genetics(clinical)

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