Research Advances in Amyotrophic Lateral Sclerosis, 2009 to 2010

被引：0
作者：
Rebecca Traub
Hiroshi Mitsumoto
Lewis P. Rowland
机构：
[1] Columbia University Medical Center,Eleanor and Lou Gehrig MDA/ALS Research Center, Neurological Institute
来源：
Current Neurology and Neuroscience Reports | 2011年 / 11卷
关键词：
Amyotrophic lateral sclerosis; Motor neuron disease; SOD1; TDP-43; TARDBP; FUS/TLS; microRNA; Stem cells; RNA interference;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease of upper and lower motor neurons that causes progressive weakness and death. The breadth of research in ALS continues to grow with exciting new discoveries in disease pathogenesis and potential future therapeutics. There is a growing list of identified mutations in familial ALS, including those in genes encoding TDP-43 and FUS/TLS, which are expanding our understanding of the role of RNA modulation in ALS pathogenesis. There is a greater appreciation for the role of glial cells in motor neuron disease. Mitochondrial dysfunction is also being shown to be critical for motor neuron degeneration. In addition to pharmacotherapy, there are promising early developments with therapeutic implications in the areas of RNA interference, stem cell therapies, viral vector—mediated gene therapy, and immunotherapy. With greater understanding of ALS pathogenesis and exciting new therapeutic technologies, there is hope for future progress in treating this disease.
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页码：67 / 77
页数：10
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[11]  
Arai T(2010)Nuclear import impairment causes cytoplasmic trans-activation response DNA-binding protein accumulation and is associated with frontotemporal lobar degeneration Brain 133 1763-1771
[12]  
Hasegawa M(2010)Transgenic rat model of neurodegeneration caused by mutation in the TDP gene PLoS Genet. 6 e1000887-5765
[13]  
Akiyama H(2005)TLS facilitates transport of mRNA encoding an actin-stabilizing protein to dendritic spines J Cell Sci 118 5755-6816
[14]  
Neumann M(2001)Identification of an RNA binding specificity for the potential splicing factor TLS J Biol Chem 276 6807-1750
[15]  
Sampathu DM(1997)TLS (FUS) binds RNA in vivo and engages in nucleo-cytoplasmic shuttling J Cell Sci 110 1741-7650
[16]  
Kwong LK(1999)Dual transforming activities of the FUS (TLS)-ERG leukemia fusion protein conferred by two N-terminal domains of FUS (TLS) Mol Cell Biol 19 7639-3729
[17]  
Yokoseki A(1994)TLS/FUS fusion domain of TLS/FUS-erg chimeric protein resulting from the t(16;21) chromosomal translocation in human myeloid leukemia functions as a transcriptional activation domain Oncogene 9 3717-8
[18]  
Shiga A(2009)Mutations in the FUS/TLS gene on chromosome 16 cause familial amyotrophic lateral sclerosis Science 323 1205-11
[19]  
Tan CF(2009)Mutations in FUS, an RNA processing protein, cause familial amyotrophic lateral sclerosis type 6 Science 323 1208-645
[20]  
Gitcho MA(2010)FUS mutations in amyotrophic lateral sclerosis: clinical, pathological, neurophysiological and genetic analysis J Neurol Neurosurg Psychiatry 81 639-1275
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