Alternative splicing

therapeutic target and tool.

Research output: Contribution to journalArticle

26 Citations (Scopus)

Abstract

Alternative splicing swells the coding capacity of the human genome, expanding the pharmacoproteome, the proteome that provides targets for therapy. Splicing, both constitutive and regulated forms, can itself be targeted by conventional and molecular therapies. This review focuses on splicing as a therapeutic target with a particular emphasis on molecular approaches. The review looks at the use of antisense oligonucleotides, which can be employed to promote skipping of constitutive exons, inhibit inappropriately activated exons, or stimulate exons weakened by mutations. Additionally this manuscript evaluates methods that reprogram RNAs using reactions that recombine RNA molecules in trans. Preliminary, but exciting, results in these areas of investigation suggest that these methods could eventually lead to treatments in heretofore intractable ailments.

Original languageEnglish
Pages (from-to)47-64
Number of pages18
JournalProgress in molecular and subcellular biology
Volume44
StatePublished - 2006
Externally publishedYes

Fingerprint

Alternative Splicing
Exons
RNA
Molecular Targeted Therapy
Antisense Oligonucleotides
Human Genome
Proteome
Therapeutics
Mutation

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Alternative splicing : therapeutic target and tool. / Garcia-Blanco, Mariano.

In: Progress in molecular and subcellular biology, Vol. 44, 2006, p. 47-64.

Research output: Contribution to journalArticle

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