Article

Chemical engineering of therapeutic siRNAs for allele-specific gene silencing <i>in vivo</i> in CNS

2022-07-02

Abstract excerpt

Small interfering RNAs (siRNAs) 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. Many genetically-defined neurodegenerative disorders are autosomal dominant favoring selective silencing of the mutant allele. In some cases, successfu...

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Literature Corpus work: 68ab2661-202e-5b64-9f5d-393d9b0bc807
DOI: 10.1101/2022.06.29.498088

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