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 …