Over a span of more than two decades, antisense strategies for gene therapy have expanded from antisense oligonucleotides (AS-ODNs) solely, to the addition of ribozymes and, more recently, to the inclusion of small interfering RNAs (siRNAs). Antisense therapeutics has also experienced its phases of high expectation, sudden disappointment, and meticulous rediscovery, while maintaining its status as a viable and effective gene therapy approach. With the discovery of RNA interference (RNAi) and development in delivery of these gene drugs, more preclinical and clinical investigations are anticipated to take place in the near future to finally fulfill the promise of antisense therapeutics in humans.
