TY - JOUR T1 - RNA Interference in Biology and Medicine JF - Pharmacological Reviews JO - Pharmacol Rev SP - 629 LP - 648 DO - 10.1124/pr.55.4.1 VL - 55 IS - 4 AU - Ollivier Milhavet AU - Devin S. Gary AU - Mark P. Mattson Y1 - 2003/12/01 UR - http://pharmrev.aspetjournals.org/content/55/4/629.abstract N2 - First discovered in plants the nematode Caenorhabditis elegans, the production of small interfering RNAs (siRNAs) that bind to and induce the degradation of specific endogenous mRNAs is now recognized as a mechanism that is widely employed by eukaryotic cells to inhibit protein production at a post-transcriptional level. The endogenous siRNAs are typically 19- to 23-base double-stranded RNA oligonucleotides, produced from much larger RNAs that upon binding to target mRNAs recruit RNases to a protein complexthat degrades the targeted mRNA. Methods for expressing siRNAs in cells in culture and in vivo using viral vectors, and for transfecting cells with synthetic siRNAs, have been developed and are being used to establish the functions of specific proteins in various cell types and organisms. RNA interference methods provide several major advantages over prior methods (antisense DNA or antibody-based techniques) for suppressing gene expression. Recent preclinical studies suggest that RNA interference technology holds promise for the treatment of various diseases. Pharmacologists have long dreamed of the ability to selectively antagonize or eliminate the function of individual proteins—RNAi technology may eventually make that dream a reality. ER -