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

• Main Authors: Raygene Martier (Author), Jolanda M. Liefhebber (Author), Ana García-Osta (Author), Jana Miniarikova (Author), Mar Cuadrado-Tejedor (Author), Maria Espelosin (Author), Susana Ursua (Author), Harald Petry (Author), Sander J. van Deventer (Author), Melvin M. Evers (Author), Pavlina Konstantinova (Author)
• Format: Book
• Published: Elsevier, 2019-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...