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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Detalles Bibliográficos
Autores principales: Martier, Raygene, Liefhebber, Jolanda M., García-Osta, Ana, Miniarikova, Jana, Cuadrado-Tejedor, Mar, Espelosin, Maria, Ursua, Susana, Petry, Harald, van Deventer, Sander J., Evers, Melvin M., Konstantinova, Pavlina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6393708/
https://www.ncbi.nlm.nih.gov/pubmed/30825670
http://dx.doi.org/10.1016/j.omtn.2019.02.001

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