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Open AccessChemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models
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 optimi...
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Article
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system
Sustained silencing of gene expression throughout the brain using small interfering RNAs (siRNAs) has not been achieved. Here we describe an siRNA architecture, divalent siRNA (di-siRNA), that supports potent,...
