Risk Factors and Emerging Therapies in Amyotrophic Lateral Sclerosis
| Autor: | Judyta K. Juranek, Jakub Juranek, Joanna Wojtkiewicz, Natalia Nowicka |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2019 |
| Předmět: |
SOD1
Disease Review Bioinformatics Catalysis Inorganic Chemistry lcsh:Chemistry Superoxide Dismutase-1 C9orf72 environmental factors medicine Animals Humans risk factors Genetic Predisposition to Disease Molecular Targeted Therapy Physical and Theoretical Chemistry Amyotrophic lateral sclerosis Genetic risk Molecular Biology lcsh:QH301-705.5 Spectroscopy C9orf72 Protein business.industry Organic Chemistry Amyotrophic Lateral Sclerosis General Medicine Genetic Therapy medicine.disease Computer Science Applications Clinical trial DNA-Binding Proteins lcsh:Biology (General) lcsh:QD1-999 Experimental medicines genetic factors ALS treatment Stem cell ALS business |
| Zdroj: | International Journal of Molecular Sciences, Vol 20, Iss 11, p 2616 (2019) International Journal of Molecular Sciences |
| ISSN: | 1422-0067 |
| Popis: | Amyotrophic lateral sclerosis (ALS) is a fatal progressive neurodegenerative disease characterized by a permanent degeneration of both upper and lower motor neurons. Many different genes and pathophysiological processes contribute to this disease, however its exact cause remains unclear. Therefore, it is necessary to understand this heterogeneity to find effective treatments. In this review, we focus on selected environmental and genetic risk factors predisposing to ALS and highlight emerging treatments in ALS therapy. Of numerous defective genes associated with ALS, we focus on four principal genes that have been identified as definite causes of ALS: the SOD1 gene, C9orf72, TDP-43, as well as the recently identified TBK1. We also provide up-to-date information on selected environmental factors that have historically been considered as key players in ALS development and pathogenesis. In parallel to our survey of known risk factors, we also discuss emerging ALS stem cell therapies and experimental medicines currently undergoing phase II and III clinical trials. |
| Databáze: | OpenAIRE |
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