Modulation of gene expression by antisense and antigene oligodeoxynucleotides and small interfering RNA

• Published in: Expert opinion on drug delivery Vol. 2; no. 1; p. 3
• Main Authors: Mahato, Ram I, Cheng, Kun, Guntaka, Ramareddy V
• Format: Journal Article
• Language: English
• Published: England 01.01.2005
• Subjects: Animals; DNA - chemistry; DNA - genetics; DNA - metabolism; Drug Carriers; Gene Expression Regulation; Gene Silencing; Gene Targeting - methods; Genetic Therapy - methods; Humans; Nucleic Acid Conformation - drug effects; Oligonucleotides, Antisense - chemistry; Oligonucleotides, Antisense - genetics; Oligonucleotides, Antisense - metabolism; Oligonucleotides, Antisense - pharmacology; Protein Biosynthesis - drug effects; RNA Interference; RNA Splicing - drug effects; RNA, Messenger - genetics; RNA, Messenger - metabolism; RNA, Small Interfering - chemistry; RNA, Small Interfering - genetics; RNA, Small Interfering - metabolism; Transcription, Genetic - drug effects
• ISSN: 1742-5247
• DOI: 10.1517/17425247.2.1.3

Antisense oligodeoxynucleotides, triplex-forming oligodeoxynucleotides and double-stranded small interfering RNAs have great potential for the treatment of many severe and debilitating diseases. Concerted efforts from both industry and academia have made significant progress in turning these nucleic acid drugs into therapeutics, and there is already one FDA-approved antisense drug in the clinic. Despite the success of one product and several other ongoing clinical trials, challenges still exist in their stability, cellular uptake, disposition, site-specific delivery and therapeutic efficacy. The principles, strategies and delivery consideration of these nucleic acids are reviewed. Furthermore, the ways to overcome the biological barriers are also discussed so that therapeutic concentrations at their target sites can be maintained for a desired period.