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

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

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...

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Main Authors: Raygene Martier, Jolanda M. Liefhebber, Ana García-Osta, Jana Miniarikova, Mar Cuadrado-Tejedor, Maria Espelosin, Susana Ursua, Harald Petry, Sander J. van Deventer, Melvin M. Evers, Pavlina Konstantinova
Format: Article
Language:English
Published: Elsevier 2019-06-01
Series:Molecular Therapy: Nucleic Acids
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253119300204

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http://www.sciencedirect.com/science/article/pii/S2162253119300204

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