Knocking down disease with siRNAs.
Knocking down expression of disease-related genes using small interfering RNAs (siRNAs) has potential for treating a variety of illnesses. This Essay will examine the opportunities for harnessing RNA interference (RNAi) for therapy, as well as the obstacles and possible ways to circumvent them.
Dykxhoorn
Derek M
DM
CBR Institute for Biomedical Research and Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA.
Lieberman
Judy
J
D000818:Animals; D016503:Drug Delivery Systems / Q000379:methods*; D020868:Gene Silencing / Q000187:drug effects; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D007239:Infections / Q000628:therapy*; D009369:Neoplasms / Q000628:therapy*; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000737:chemistry / Q000235:genetics / Q000378:metabolism / Q000493:pharmacokinetics / Q000627:therapeutic use
D034741:RNA, Small Interfering