Journal
NEURODEGENERATIVE DISEASE MANAGEMENT
Volume 4, Issue 6, Pages 417-437Publisher
FUTURE MEDICINE LTD
DOI: 10.2217/NMT.14.36
Keywords
ALS; ALS therapeutics; C9ORF72; repeat expansion; RNA toxicity; TDP-43
Categories
Funding
- Brain Science Institute
- NINDS [RO1 NS085207]
- Judith and Jean Pape Adams Charitable Foundation
- ALS Association
- Maryland Stem Cell Research Fund
- William and Ella Owens Medical Research Foundation
- National Science Foundation Graduate Research Fellowship Award
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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.
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