The orchestration of brain function requires complex gene regulatory networks that are modulated, in part, by microRNAs (miRNAs). These noncoding RNAs associate with argonaute (Ago) proteins in order to direct posttranscriptional gene suppression via base pairing with target transcripts. In order to better understand how miRNAs contribute to… (More)
Huntington's disease (HD) is caused by a polyglutamine tract expansion in huntingtin (HTT). Despite HTTs ubiquitous expression, there is early and robust vulnerability in striatum, the cause of which is poorly understood. Here, we provide evidence that impaired striatal mTORC1 activity underlies varied metabolic and degenerative phenotypes in HD brain and… (More)
Single-stranded RNAs interact with components of the RNA interference pathway to reduce the expression of target mRNAs. Now, Lima et al. and Yu et al. show that, with extensive chemical modifications, small single-stranded RNAs can robustly induce gene silencing with efficacy similar to their double-stranded counterparts.