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An NLRP3 Mutation Causes Arthropathy and Osteoporosis in Humanized Mice

Title: An NLRP3 Mutation Causes Arthropathy and Osteoporosis in Humanized Mice
Authors: Snouwaert, J.N.; Nguyen, M.; Repenning, P.W.; Dye, R.; Livingston, E.W.; Kovarova, M.; Moy, S.S.; Brigman, B.E.; Bateman, T.A.; Ting, J.P.-Y.; Koller, B.H.
Source: Cell Reports, 17(11)
Publisher Information: Elsevier B.V.
Publication Year: 2016
Collection: Carolina Digital Repository (UNC - University of North Carolina)
Subject Terms: autoinflammatory; NOMID; endotoxic shock; familial cold autoinflammatory syndrome; inflammasome; IL-18; arthritis; syntenic replacement; meningitis; Muckle-Wells syndrome; cryopyrinopathies
Description: The NLRP3 inflammasome plays a critical role in host defense by facilitating caspase I activation and maturation of IL-1β and IL-18, whereas dysregulation of inflammasome activity results in autoinflammatory disease. Factors regulating human NLRP3 activity that contribute to the phenotypic heterogeneity of NLRP3-related diseases have largely been inferred from the study of Nlrp3 mutant mice. By generating a mouse line in which the NLRP3 locus is humanized by syntenic replacement, we show the functioning of the human NLRP3 proteins in vivo, demonstrating the ability of the human inflammasome to orchestrate immune reactions in response to innate stimuli. Humanized mice expressing disease-associated mutations develop normally but display acute sensitivity to endotoxin and develop progressive and debilitating arthritis characterized by granulocytic infiltrates, elevated cytokines, erosion of bones, and osteoporosis. This NLRP3-dependent arthritis model provides a platform for testing therapeutic reagents targeting the human inflammasome.
Document Type: article in journal/newspaper
Language: English
Relation: https://cdr.lib.unc.edu/downloads/4x51ht32g?file=thumbnail; https://cdr.lib.unc.edu/downloads/4x51ht32g
DOI: 10.17615/9799-1p56
Availability: https://doi.org/10.17615/9799-1p56; https://cdr.lib.unc.edu/downloads/4x51ht32g?file=thumbnail; https://cdr.lib.unc.edu/downloads/4x51ht32g
Rights: http://rightsstatements.org/vocab/InC/1.0/
Accession Number: edsbas.1ABFAA67
Database: BASE