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.
Keywords: Amyotrophic lateral sclerosis (ALS), gene expression, messenger RNA (mRNA), micro RNA (miRNA), RNA interference (RNAi), short (or small) hair-pin RNAs (shRNA), short (or small) interfering RNA (siRNA), superoxide dismutase (SOD), Thomas Tuschl's group, silencer RNAs
Current Drug Metabolism
Title: RNA Interference and Amyotrophic Lateral Sclerosis
Volume: 12 Issue: 7
Author(s): Albert A. Rizvanov, Sukru Gulluoglu, Mehmet E. Yalvac, Andras Palotas and Rustem R. Islamov
Affiliation:
Keywords: Amyotrophic lateral sclerosis (ALS), gene expression, messenger RNA (mRNA), micro RNA (miRNA), RNA interference (RNAi), short (or small) hair-pin RNAs (shRNA), short (or small) interfering RNA (siRNA), superoxide dismutase (SOD), Thomas Tuschl's group, silencer RNAs
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.
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Cite this article as:
A. Rizvanov Albert, Gulluoglu Sukru, E. Yalvac Mehmet, Palotas Andras and R. Islamov Rustem, RNA Interference and Amyotrophic Lateral Sclerosis, Current Drug Metabolism 2011; 12 (7) . https://dx.doi.org/10.2174/138920011796504464
DOI https://dx.doi.org/10.2174/138920011796504464 |
Print ISSN 1389-2002 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5453 |
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