Aberrant RNA homeostasis in amyotrophic lateral sclerosis: potential for new therapeutic targets?
Autor: | Rita Sattler, Jonathan C. Grima, Christopher J. Donnelly |
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Rok vydání: | 2014 |
Předmět: |
Biology
medicine.disease_cause TARDBP Article C9orf72 medicine Animals Homeostasis Humans RNA Processing Post-Transcriptional Amyotrophic lateral sclerosis Gene Mutation C9orf72 Protein Amyotrophic Lateral Sclerosis Proteins RNA medicine.disease DNA-Binding Proteins RNA-Binding Protein FUS Neurology (clinical) Trinucleotide repeat expansion Neuroscience |
Zdroj: | Neurodegenerative Disease Management. 4:417-437 |
ISSN: | 1758-2032 1758-2024 |
Popis: | SUMMARY 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. |
Databáze: | OpenAIRE |
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