TY - JOUR
T1 - Rodent models of TDP-43
T2 - Recent advances
AU - Tsao, William
AU - Jeong, Yun Ha
AU - Lin, Sophie
AU - Ling, Jonathan
AU - Price, Donald L.
AU - Chiang, Po Min
AU - Wong, Philip C.
N1 - Funding Information:
The authors would like to thank Xiu Shan, Susan Aja, Venette Nehus and Frances Davenport to their contributions to some of the original TDP-43 work cited in this review. Aspects of this work were supported by grants from the National Institute of Neurological Disorders and Stroke Grant R01 NS41438 , the Muscular Dystrophy Association , The Robert Packard Center for ALS Research and The Johns Hopkins Neuropathology Gift Fund .
PY - 2012/6/26
Y1 - 2012/6/26
N2 - 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.
AB - 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.
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U2 - 10.1016/j.brainres.2012.04.031
DO - 10.1016/j.brainres.2012.04.031
M3 - Review article
C2 - 22608070
AN - SCOPUS:84862118369
VL - 1462
SP - 26
EP - 39
JO - Molecular Brain Research
JF - Molecular Brain Research
SN - 0006-8993
ER -