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- W1978214082 abstract "Last year's Nobel Prize in Physiology or Medicine was won by Andrew Fire and Craig Mello for their discovery of RNA interference, an evolutionarily conserved cellular pathway that regulates endogenous gene expression. In this Review, Gonzalez and Paulson discuss how RNA interference is being exploited experimentally to suppress the expression of dominantly acting genes that cause incurable neurodegenerative disorders, and highlight the questions that need to be answered before human clinical trials of this technology can commence. As an evolutionarily conserved cellular pathway to regulate endogenous gene expression, RNA interference (RNAi) has been implicated in diverse biological processes. Biologists now routinely exploit this cellular pathway to suppress virtually any target gene in a sequence-specific manner, including dominantly acting genes that cause incurable neurodegenerative disorders. The development of RNAi as potential therapy for such diseases has generated considerable interest, partly because of the success of early studies of therapeutic RNAi in rodent models for a range of neurodegenerative diseases. In this article, we review the progress of RNAi therapy to date, and assess the challenges ahead for the application of such therapy to neurodegenerative diseases. We discuss the various strategies that might be used to achieve this goal, outline the preclinical studies that have already been completed, and highlight the experimental questions that need to be answered before human clinical trials can begin." @default.
- W1978214082 created "2016-06-24" @default.
- W1978214082 creator A5005213034 @default.
- W1978214082 creator A5055180300 @default.
- W1978214082 date "2007-07-01" @default.
- W1978214082 modified "2023-10-18" @default.
- W1978214082 title "Technology Insight: therapeutic RNA interference—how far from the neurology clinic?" @default.
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- W1978214082 doi "https://doi.org/10.1038/ncpneuro0551" @default.
- W1978214082 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/17611488" @default.
- W1978214082 hasPublicationYear "2007" @default.
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