期刊
BRAIN RESEARCH
卷 1462, 期 -, 页码 26-39出版社
ELSEVIER
DOI: 10.1016/j.brainres.2012.04.031
关键词
TDP-43; Transgenic; Conditional knockout
资金
- National Institute of Neurological Disorders and Stroke [R01 NS41438]
- Muscular Dystrophy Association
- Robert Packard Center for ALS Research
- Johns Hopkins Neuropathology Gift Fund
Recently, missense mutations in the gene TARDBP encoding TDP-43 have been linked to familial ALS. The discovery of genes encoding these RNA binding proteins, such as TDP-43 and FUS/TLS, raised the notion that altered RNA metabolism is a major factor underlying the pathogenesis of ALS. To begin to unravel how mutations in TDP-43 cause dysfunction and death of motor neurons, investigators have employed both gain- and loss-of-function studies in rodent model systems. Here, we will summarize major findings from the initial sets of TDP-43 transgenic and knockout rodent models, identify their limitations, and point to future directions toward clarification of disease mechanism(s) and testing of therapeutic strategies that ultimately may lead to novel therapy for this devastating disease. This article is part of a Special Issue entitled RNA-Binding Proteins. (c) 2012 Elsevier B.V. All rights reserved.
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