Making antisense of splicing

Mariano A. Garcia-Blanco

Research output: Contribution to journalReview articlepeer-review

19 Scopus citations


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
Issue number5
StatePublished - Oct 2005
Externally publishedYes


  • 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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