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

• मुख्य लेखकों: Raygene Martier (लेखक), Jolanda M. Liefhebber (लेखक), Jana Miniarikova (लेखक), Tom van der Zon (लेखक), Jolanda Snapper (लेखक), Iris Kolder (लेखक), Harald Petry (लेखक), Sander J. van Deventer (लेखक), Melvin M. Evers (लेखक), Pavlina Konstantinova (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2019-03-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...