Journal
CURRENT GENE THERAPY
Volume 23, Issue 2, Pages 148-162Publisher
BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/1566523223666221108113330
Keywords
ALS; SOD1; C9ORF72; TDP-43; FUS; putative targets; mutations; suppressor
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Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive paralysis and respiratory failure. While the disease mechanisms are not fully understood, around 40 genes have been identified as ALS causative genes, with mutations in key genes leading to various cellular dysfunctions. Recent studies have shown that repairing a single cellular pathway can restore other dysfunctions in ALS. This review aims to identify potential therapeutic targets and highlight the importance of a single suppressor in reducing multiple symptoms of ALS.
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that leads to death by progressive paralysis and respiratory failure within 2-4 years of onset. About 90-95% of ALS cases are sporadic (sALS), and 5-10% are inherited through family (fALS). Though the mechanisms of the disease are still poorly understood, so far, approximately 40 genes have been reported as ALS causative genes. The mutations in some crucial genes, like SOD1, C9ORF72, FUS, and TDP-43, are majorly associated with ALS, resulting in ROS-associated oxidative stress, excitotoxicity, protein aggregation, altered RNA processing, axonal and vesicular trafficking dysregulation, and mitochondrial dysfunction. Recent studies show that dysfunctional cellular pathways get restored as a result of the repair of a single pathway in ALS. In this review article, our aim is to identify putative targets for therapeutic development and the importance of a single suppressor to reduce multiple symptoms by focusing on important mutations and the phenotypic suppressors of dysfunctional cellular pathways in crucial genes as reported by other studies.
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