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- W4300594197 abstract "Abstract Small interfering RNAs are a new class of drugs, exhibiting sequence-driven, potent, and sustained silencing of gene expression in vivo. We recently demonstrated that siRNA chemical architectures can be optimized to provide efficient delivery to the CNS, enabling development of CNS-targeted therapeutics. Many genetically-defined neurodegenerative disorders are dominant, favoring selective silencing of the mutant allele. In some cases, successfully targeting the mutant allele requires targeting single nucleotide polymorphism (SNP) heterozygosities. Here, we use Huntington’s disease (HD) as a model. The optimized compound exhibits selective silencing of mutant huntingtin protein in patient-derived cells and throughout the HD mouse brain, demonstrating SNP-based allele-specific RNAi silencing of gene expression in vivo in the CNS. Targeting a disease-causing allele using RNAi-based therapies could be helpful in a range of dominant CNS disorders where maintaining wild-type expression is essential." @default.
- W4300594197 created "2022-10-03" @default.
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- W4300594197 date "2022-10-03" @default.
- W4300594197 modified "2023-10-14" @default.
- W4300594197 title "Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models" @default.
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- W4300594197 doi "https://doi.org/10.1038/s41467-022-33061-x" @default.
- W4300594197 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/36192390" @default.
- W4300594197 hasPublicationYear "2022" @default.
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