Article
Murine knockin model for progranulin-deficient frontotemporal dementia with nonsense-mediated mRNA decay.
Proceedings of the National Academy of Sciences of the United States of America - 20 Mar 2018
Nguyen Andrew D, Nguyen Thi A, Zhang Jiasheng, Devireddy Swathi, Zhou Ping, Karydas Anna M, Xu Xialian, Miller Bruce L, Rigo Frank, Ferguson Shawn M, Huang Eric J, Walther Tobias C, Farese Robert V
Abstract excerpt
Frontotemporal dementia (FTD) is the most common neurodegenerative disorder in individuals under age 60 and has no treatment or cure. Because many cases of FTD result from GRN nonsense mutations, an animal model for this type of mutation is highly desirable for understanding pathogenesis and testing therapies. Here, we generated and characterized GrnR493X knockin mice, which model the most common human GRN...
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