Short-term and long-term modulation of gene expression by antisense therapeutics

Abstract

To achieve effective modulation of gene expression by antisense oligonucleotides, novel oligonucleotide chemistries that do not promote RNase H degradation of target RNA are needed. In addition to short-term oligonucleotide effects, long-term gene regulation can be accomplished by intracellularly expressed antisense RNAs delivered by viral vectors.