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Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy

11 Feb 2019

Abstract excerpt

A hexanucleotide GGGGCC expansion in intron 1 of chromosome 9 open reading frame 72 (C9orf72) gene is the most frequent cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The corresponding repeat-containing sense and antisense transcripts cause a gain of toxicity through the accumulation of RNA foci in the nucleus and deposition of dipeptide-repeat (DPR) proteins in the cytoplasm of...

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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: 30825670
DOI: 10.1016/j.omtn.2019.02.001

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Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene TherapyPMID 30825670Read ↗

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