MODIFICATION OF PRE-MESSENGER-RNA SPLICING BY ANTISENSE OLIGONUCLEOTIDES

Antisene oligonucleotides have been extensively studied as agents that inhibit the expression of undesirable genes in a sequence specific ma nner. Results reviewed in this article show that antisene oligonucleot ides can also restore the expression of genes inactivated by mutations causing genetic disease. In this novel application, antisene oligonuc leotides 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 g ene.