Dysregulation of RNA Mediated Gene Expression in Motor Neuron Diseases

Author(s): Inês do Carmo G. Gonçalves, Wiebke A. Rehorst, Min Jeong Kye

Journal Name: CNS & Neurological Disorders - Drug Targets
(Formerly Current Drug Targets - CNS & Neurological Disorders)

Volume 15 , Issue 8 , 2016

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Recent findings indicate an important role for RNA-mediated gene expression in motor neuron diseases, including ALS (amyotrophic lateral sclerosis) and SMA (spinal muscular atrophy). ALS, also known as Lou Gehrig’s disease, is an adult-onset progressive neurodegenerative disorder, whereby SMA or “children’s Lou Gehrig’s disease” is considered a pediatric neurodevelopmental disorder. Despite the difference in genetic causes, both ALS and SMA share common phenotypes; dysfunction/loss of motor neurons that eventually leads to muscle weakness and atrophy. With advanced techniques in molecular genetics and cell biology, current data suggest that these two distinct motor neuron diseases share more than phenotypes; ALS and SMA have similar cellular pathological mechanisms including mitochondrial dysfunction, oxidative stress and dysregulation in RNA-mediated gene expression. Here, we will discuss the current findings on these two diseases with specific focus on RNA-mediated gene regulation including miRNA expression, pre-mRNA processing and RNA binding proteins.

Keywords: Amyotrophic lateral sclerosis, microRNA, motor neuron disease, mRNA translation, RNA binding proteins, SMN, Spinal muscular atrophy, TDP-43/FUS.

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Article Details

Year: 2016
Published on: 08 September, 2016
Page: [887 - 895]
Pages: 9
DOI: 10.2174/1871527315666160815164808
Price: $65

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