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• W2040982254 abstract "Genetic diseases that are accompanied by central nervous system involvement are often fatal. Among these are the autosomal dominant neurogenetic diseases caused by nucleotide repeat expansion. For example, Huntington's disease (HD) and spinal cerebellar ataxia are caused by expansion of a tract of CAGs encoding glutamine. In HD and the other CAG-repeat expansion diseases, the expansion is in the coding region. Myotonic dystrophy is caused by repeat expansions of CUG or CCTG in noncoding regions, and the mutant RNA is disease causing. Treatments for these disorders are limited to symptomatic intervention. RNA interference (RNAi), which is a method for inhibiting target gene expression, provides a unique tool for therapy by attacking the fundamental problem directly. In this review, we describe briefly several representative disorders and their respective molecular targets, and methods to accomplish therapeutic RNAi. Finally, we summarize studies performed to date." @default.
• W2040982254 created "2016-06-24" @default.
• W2040982254 creator A5045163747 @default.
• W2040982254 creator A5088113414 @default.
• W2040982254 date "2005-10-20" @default.
• W2040982254 modified "2023-10-10" @default.
• W2040982254 title "RNAi: a potential therapy for the dominantly inherited nucleotide repeat diseases" @default.
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• W2040982254 doi "https://doi.org/10.1038/sj.gt.3302664" @default.
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