Applying small RNA molecules to the directed treatment of human diseases: realizing the potential.
Small interfering RNAs (siRNA) and microRNAs (miRNA) are gaining considerable attention in the pharmaceutical and biotechnology industries, as research has revealed their likely clinical and agricultural applications. The capacity of siRNAs to dramatically and specifically reduce the expression of targeted genes has spawned multiple clinical trials to establish the therapeutic potential of small RNAs targeting viral, cancer and other disease-related genes. The successful application of siRNAs will enable the development of therapeutic applications based on miRNAs that have been observed to contribute to a variety of human diseases. This article reviews advances that have been made to apply small RNAs as therapeutics.
Pappas
Todd C
TC
Asuragen, Inc., 2150 Woodward Street, Suite 100, Austin, TX 78744, USA.
Bader
Andreas G
AG
Andruss
Bernard F
BF
Brown
David
D
Ford
Lance P
LP
D000818:Animals; D002986:Clinical Trials as Topic / Q000639:trends*; D020868:Gene Silencing / Q000502:physiology; D018390:Gene Targeting / Q000639:trends*; D006801:Humans; D035683:MicroRNAs / Q000037:antagonists & inhibitors / Q000096:biosynthesis / Q000378:metabolism*; D034741:RNA, Small Interfering / Q000096:biosynthesis / Q000235:genetics* / Q000627:therapeutic use*
D035683:MicroRNAs; D034741:RNA, Small Interfering