The promises and pitfalls of RNA-interference-based therapeutics.
The discovery that gene expression can be controlled by the Watson-Crick base-pairing of small RNAs with messenger RNAs containing complementary sequence - a process known as RNA interference - has markedly advanced our understanding of eukaryotic gene regulation and function. The ability of short RNA sequences to modulate gene expression has provided a powerful tool with which to study gene function and is set to revolutionize the treatment of disease. Remarkably, despite being just one decade from its discovery, the phenomenon is already being used therapeutically in human clinical trials, and biotechnology companies that focus on RNA-interference-based therapeutics are already publicly traded.
Castanotto
Daniela
D
Department of Molecular Biology and City of Hope Graduate School of Biological Sciences, Beckman Research Institute of the City of Hope, Duarte, California 91010, USA.
Rossi
John J
JJ
D000818:Animals; D002986:Clinical Trials as Topic; D055785:Gene Knockdown Techniques; D015316:Genetic Therapy / Q000009:adverse effects / Q000639:trends*; D006801:Humans; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000009:adverse effects / Q000235:genetics / Q000627:therapeutic use*
D034741:RNA, Small Interfering