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Article

Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models.

Nature communications - 3 Oct 2022

Conroy Faith, Miller Rachael, Alterman Julia F, Hassler Matthew R, Echeverria Dimas, Godinho Bruno M D C, Knox Emily G, Sapp Ellen, Sousa Jaquelyn, Yamada Ken, Mahmood Farah, Boudi Adel, Kegel-Gleason Kimberly, DiFiglia Marian, Aronin Neil, Khvorova Anastasia, Pfister Edith L

Abstract excerpt

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...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 36192390
DOI: 10.1038/s41467-022-33061-x
PMCID: PMC9530163

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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