Toxicity in mice expressing short hairpin RNAs gives new insight into RNAi.
Short hairpin RNAs can provide stable gene silencing via RNA interference. Recent studies have shown toxicity in vivo that appears to be related to saturation of the endogenous microRNA pathway. Will these findings limit the therapeutic use of such hairpins?
Snøve
Ola
O
Division of Molecular Biology, Beckman Research Institute of the City of Hope, 1450 East Duarte Road, Duarte, CA 91101, USA.
Rossi
John J
JJ
D000818:Animals; D020868:Gene Silencing; D015316:Genetic Therapy / Q000009:adverse effects*; D005822:Genetic Vectors / Q000633:toxicity; D007109:Immunity; D051379:Mice; D035683:MicroRNAs / Q000378:metabolism / Q000627:therapeutic use / Q000633:toxicity; D034622:RNA Interference; D012323:RNA Processing, Post-Transcriptional; D034741:RNA, Small Interfering / Q000378:metabolism / Q000627:therapeutic use / Q000633:toxicity*
D035683:MicroRNAs; D034741:RNA, Small Interfering