RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia

By Haibin Xia, Q. Mao, S. Eliason, S. Harper, Inês H Martins, H. Orr, H. Paulson, Linda C. Yang, R. Kotin, B. Davidson. Nature Medicine, 2004

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The promises and pitfalls of RNA-interference-based therapeutics (2009, neutral): “For instance, direct brain injection of an adenoviral vector expressing a shRNA directed against the mRNA encoding the polyQ-harbouring SCA1-encoding transcript of spinocerebellar ataxia type 1 was shown to be an effective treatment in a mouse model of this disorde”
RNAi therapeutics: a potential new class of pharmaceutical drugs (2006, background): “as well as in animal models of spinocerebellar ataxi”
Current prospects for RNA interference-based therapies (2011, background): “For example, vectors expressing therapeutic RNAi improved disease phenotypes for many months in preclinical studies in rodent models of polyglutamine repeat diseases [108] [109] [110] [111] [112] [113] , amyotrophic lateral sclerosis [114] [115] [116] , Parkinson's disease 117 and Alzheimer's diseas”
MicroRNA dysregulation in cancer: diagnostics, monitoring and therapeutics. A comprehensive review (2012, neutral): “…the expression can be enforced by a viral vector with Pol III promoter, including U6, H1 and tRNA (Kawasaki & Taira, 2003; Tiscornia et al, 2003; Xia et al, 2004), upstream an artiﬁcial short hairpin RNA (shRNA), which bypasses Drosha processing but it is cleaved and loaded into miRISC by Dicer.”