Modification of pre-mRNA splicing by antisense oligonucleotides.

R Kole

doi:10.18388/abp.1997_4418

Modification of pre-mRNA splicing by antisense oligonucleotides.

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

DOI: https://doi.org/10.18388/abp.1997_4418

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.

Published

1997-06-30

Issue

Vol. 44 No. 2 (1997)

Section

Articles

Acta Biochimica Polonica is an OpenAccess quarterly and publishes four issues a year. All contents are distributed under the Creative Commons Attribution-ShareAlike 4.0 International (CC BY 4.0) license. Everybody may use the content following terms: Attribution — You must give appropriate credit, provide a link to the license, and indicate if changes were made. You may do so in any reasonable manner, but not in any way that suggests the licensor endorses you or your use.