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• W2094973973 endingPage "64" @default.
• W2094973973 startingPage "50" @default.
• W2094973973 abstract "Huntington's disease (HD) is an incurable genetic neurodegenerative disorder that leads to motor and cognitive decline. It is caused by an expanded polyglutamine tract within the Huntingtin (HTT) gene, which translates into a toxic mutant HTT (muHTT) protein. Although no cure has yet been discovered, novel therapeutic strategies, such as RNA interference (RNAi), antisense oligonucleotides (ASOs), ribozymes, DNA enzymes, and genome-editing approaches, aimed at silencing or repairing the muHTT gene hold great promise. Indeed, several preclinical studies have demonstrated the utility of such strategies to improve HD neuropathology and symptoms. In this review, we critically summarise the main advances and limitations of each gene-silencing technology as an effective therapeutic tool for the treatment of HD." @default.
• W2094973973 created "2016-06-24" @default.
• W2094973973 creator A5010843022 @default.
• W2094973973 creator A5039402697 @default.
• W2094973973 creator A5078087258 @default.
• W2094973973 creator A5084928512 @default.
• W2094973973 date "2015-01-01" @default.
• W2094973973 modified "2023-10-18" @default.
• W2094973973 title "Delivering a disease-modifying treatment for Huntington's disease" @default.
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