期刊
EXPERT OPINION ON BIOLOGICAL THERAPY
卷 8, 期 7, 页码 969-978出版社
TAYLOR & FRANCIS LTD
DOI: 10.1517/14712598.8.7.969
关键词
amyotropic lateral sclerosis; frontotemporal dementia; progranulin; TAR DNA binding protein-43 (TDP-43); valosin-containing protein
资金
- NIA NIH HHS [R01 AG026251, R01 AG026251-02, R01-AG-026251-01] Funding Source: Medline
Background: The number of neurodegenerative diseases associated with pathological aggregates of transactivation response element (TAR)-DNA-binding protein 43 (TDP-43) has increased, leading to the new designation 'TDP-43 proteinopathy.' Biochemically, TDP-43 proteinopathies are characterized by decreased solubility, hyperphosphorylation, and cleavage of TDP-43 into 25- and 35-kDa fragments, and by altered cellular localization. Objective: This review summarizes research characterizing the distribution of TDP-43 pathology in human postmortem brain tissue and discusses possible therapeutic strategies based on genetic and in vitro studies. Methods: We reviewed recent studies of TDP-43 proteinopathy. Results/conclusion: Given that several different mutations can lead to TDP-43 proteinopathies, including Mutations in progranulin and valosin-containing protein, research is needed to decipher and potentially exploit the link between these mutations and TDP-43 pathology.
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