Published on Jan. 1, 2014 in Arch Biochem Biophys volume 4(6).

PubMed ID: 25531686

DOI: 10.2217/nmt.14.36

Abstract:

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disordercharacterized by progressive motor neuron degeneration. The disease pathogenesis ismultifaceted in that multiple cellular and molecular pathways have been identifiedas contributors to the disease progression. Consequently, numerous therapeutictargets have been pursued for clinical development, unfortunately with littlesuccess. The recent discovery of mutations in RNA modulating genes such asTARDBP/TDP-43, FUS/TLS or C9ORF72 changed our understanding of neurodegenerativemechanisms in ALS and introduced the role of dysfunctional RNA processing as asignificant contributor to disease pathogenesis. This article discusses the latestfindings on such RNA toxicity pathways in ALS and potential novel therapeuticapproaches.