Open AccessModification of pre-mRNA splicing by antisense oligonucleotides.
Author(s) -
Ryszard Kole
Publication year - 1997
Publication title -
acta biochimica polonica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.452
H-Index - 78
eISSN - 1734-154X
pISSN - 0001-527X
DOI - 10.18388/abp.1997_4418
Subject(s) - oligonucleotide , rna splicing , spliceosome , gene , splice , biology , genetics , precursor mrna , computational biology , sequence (biology) , alternative splicing , exon , microbiology and biotechnology , rna
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.