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Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs

1 Aug 2024

Abstract excerpt

A hexanucleotide (G4C2) repeat expansion (HRE) within intron one of C9ORF72 is the leading genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). C9ORF72 haploinsufficiency, formation of RNA foci, and production of dipeptide repeat (DPR) proteins have been proposed as mechanisms of disease. Here, we report the first example of disease-modifying siRNAs for C9ORF72 driven ALS/FTD....

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Amyotrophic Lateral Sclerosis Research
Neurogenetic and Muscular Disorders Research
RNA Research and Splicing

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 39233852
DOI: 10.1016/j.omtn.2024.102291

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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