title: Modification of pre-mRNA splicing by antisense oligonucleotides.
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.
authors.affiliation: Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill 27599, USA. kole@med.unc.edu
authors.forename: R
authors.initials: R
authors.lastname: Kole
mesh_terms: D000818:Animals; D015870:Gene Expression / Q000187:drug effects; D006367:HeLa Cells; D006801:Humans; D016376:Oligonucleotides, Antisense / Q000494:pharmacology*; D012322:RNA Precursors / Q000235:genetics / Q000378:metabolism*; D012326:RNA Splicing / Q000187:drug effects*; D012333:RNA, Messenger / Q000235:genetics / Q000378:metabolism*
chemical_list: D016376:Oligonucleotides, Antisense; D012322:RNA Precursors; D012333:RNA, Messenger