title: RNA interference and amyotrophic lateral sclerosis.
abstract: Amyotrophic lateral sclerosis (ALS) is a debilitating neuro-degenerative disorder characterized by progressive loss of motor neurons. The etiology and molecular pathogenesis of cell death in most sub-types of the disease are largely unknown. The best documented cause of moto-neuron degeneration is the mutation in the superoxide dismutase-1 (SOD1) gene, which occurs in 10% of the familial forms of ALS. Discovery of RNA interference (RNAi), which plays an important role in the regulation of gene expression, has proven to be a powerful tool to study the pathogenesis and to develop innovative treatment options for hereditary diseases, including familial variants of ALS. This review summarizes current research advances in RNAi in relation to ALS.
authors: Department of Genetics, Faculty of Biology and Soil Sciences, Kazan (Volga Region) Federal University, ul. Kremlevskaya 18, R-420008 Kazan, Russia.
authors: Albert A
authors: AA
authors: Rizvanov
authors: Sukru
authors: S
authors: Gulluoglu
authors: Mehmet E
authors: ME
authors: Yalvaç
authors: András
authors: A
authors: Palotás
authors: Rustem R
authors: RR
authors: Islamov
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000201:enzymology / Q000235:genetics* / Q000628:therapy*; D000818:Animals; D016923:Cell Death / Q000502:physiology; D015316:Genetic Therapy / Q000639:trends*; D006801:Humans; D009154:Mutation / Q000235:genetics; D034622:RNA Interference / Q000502:physiology*; D013482:Superoxide Dismutase / Q000235:genetics; D000072105:Superoxide Dismutase-1
chemical_list: C000606290:SOD1 protein, human; D013482:Superoxide Dismutase; D000072105:Superoxide Dismutase-1