Aberrant RNA homeostasis in amyotrophic lateral sclerosis: potential for new therapeutic targets?
Document Type
Article
Abstract
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by progressive motor neuron degeneration. The disease pathogenesis is multifaceted in that multiple cellular and molecular pathways have been identified as contributors to the disease progression. Consequently, numerous therapeutic targets have been pursued for clinical development, unfortunately with little success. The recent discovery of mutations in RNA modulating genes such as TARDBP/TDP-43, FUS/TLS or C9ORF72 changed our understanding of neurodegenerative mechanisms in ALS and introduced the role of dysfunctional RNA processing as a significant contributor to disease pathogenesis. This article discusses the latest findings on such RNA toxicity pathways in ALS and potential novel therapeutic approaches.
Keywords
ALS, ALS therapeutics, C9ORF72, repeat expansion, RNA toxicity, TDP-43
Publication Date
1-1-2014
Publication Title
Neurodegenerative disease management
E-ISSN
17582032
Volume
4
Issue
6
First Page
417
Last Page
437
PubMed ID
25531686
Digital Object Identifier (DOI)
10.2217/nmt.14.36
Recommended Citation
Donnelly, Christopher J.; Grima, Jonathan C.; and Sattler, Rita, "Aberrant RNA homeostasis in amyotrophic lateral sclerosis: potential for new therapeutic targets?" (2014). Translational Neuroscience. 1382.
https://scholar.barrowneuro.org/neurobiology/1382