Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients

• Main Authors: Raygene Martier (Author), Jolanda M. Liefhebber (Author), Jana Miniarikova (Author), Tom van der Zon (Author), Jolanda Snapper (Author), Iris Kolder (Author), Harald Petry (Author), Sander J. van Deventer (Author), Melvin M. Evers (Author), Pavlina Konstantinova (Author)
• Format: Book
• Published: Elsevier, 2019-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

The most common pathogenic mutation in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) is an intronic GGGGCC (G4C2) repeat in the chromosome 9 open reading frame 72 (C9orf72) gene. Cellular toxicity due to RNA foci and dipeptide repeat (DPR) proteins produced by the sense and a...