The therapeutic potential of RNA interference.
In recent years, we have witnessed the discovery of a new mechanism of gene regulation called RNA interference (RNAi), which has revitalized interest in the development of nucleic acid-based technologies for therapeutic gene suppression. This review focuses on the potential therapeutic use of RNAi, discussing the theoretical advantages of RNAi-based therapeutics over previous technologies as well as the challenges involved in developing RNAi for clinical use. Also reviewed, are the in vivo proof-of principle experiments that provide the preclinical justification for the continued development of RNAi-based therapeutics.
Uprichard
Susan L
SL
Department of Molecular and Experimental Medicine, The Scripps Research Institute, 10550 N. Torrey Pines Road, SBR10, La Jolla, CA 92037, USA. sluprich@scripps.edu
D000818:Animals; D000970:Antineoplastic Agents / Q000494:pharmacology; D017209:Apoptosis; D001709:Biotechnology; D016503:Drug Delivery Systems; D004345:Drug Industry; D005128:Eye Diseases / Q000628:therapy; D005786:Gene Expression Regulation; D015967:Gene Expression Regulation, Viral; D005821:Genetic Techniques; D006801:Humans; D007249:Inflammation / Q000628:therapy; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000378:metabolism; D012367:RNA, Viral / Q000235:genetics*
D000970:Antineoplastic Agents; D034741:RNA, Small Interfering; D012367:RNA, Viral