Acta Biochim Pol. 1997;44(2):231-7.

Modification of pre-mRNA splicing by antisense oligonucleotides.

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Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill 27599, USA. kole@med.unc.edu

Abstract

Antisene oligonucleotides have been extensively studied as agents that inhibit the expression of undesirable genes in a sequence specific manner. Results reviewed in this article show that antisene oligonucleotides can also restore the expression of genes inactivated by mutations causing genetic diseases. In this novel application, antisene oligonucleotides block aberrant splice sites created by the mutations, forcing the spliceosomes to form at correct splice sites, thus restoring the proper splicing pathway and consequently the activity of the damaged gene.

PMID:: 9360712; [PubMed - indexed for MEDLINE]

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Repair of thalassemic human beta-globin mRNA in mammalian cells by antisense oligonucleotides. [Proc Natl Acad Sci U S A. 1996]
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Cited by 2 PubMed Central articles

hnRNP L regulates the tumorigenic capacity of lung cancer xenografts in mice via caspase-9 pre-mRNA processing. [J Clin Invest. 2010]
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