Pulmonary function and pathology in hydroxypropyl-beta-cyclodextin-treated and untreated Npc1⁻/⁻ mice.
Document Type
Article
Abstract
Lung dysfunction is an important part of the pathology of the neurodegenerative disorder, Niemann-Pick C1 (NPC1). We have studied the pulmonary disease in the Npc1(NIH/NIH) mouse model. On histology, we find large numbers of alveolar foamy macrophages but no alveolar proteinosis. Lung weight as percent of body weight was markedly increased; using the flexiVent small animal ventilator (SCIREQ, Inc.), we find inspiratory capacity, elastance and hysterisivity to be increased while resistance was not changed. Cholesterol measurements show a doubling of lung cholesterol levels. Collagen is also increased. Treatment of Npc1(-/-) mice with hydroxypropyl-β-cyclodextrin (HPBCD), despite efficacious effects in brain and liver, results in little difference from age-matched controls (using a CNS-expressed transgene to extend the life expectancy of the Npc1(-/-) mice) for these variables.
Medical Subject Headings
2-Hydroxypropyl-beta-cyclodextrin; Animals; Cholesterol; Disease Models, Animal; Lung; Mice; Mice, Inbred BALB C; Mice, Knockout; Niemann-Pick Disease, Type C; Respiratory Function Tests; beta-Cyclodextrins
Publication Date
6-1-2011
Publication Title
Molecular genetics and metabolism
E-ISSN
1096-7206
Volume
103
Issue
2
First Page
142
Last Page
147
PubMed ID
21459030
Digital Object Identifier (DOI)
10.1016/j.ymgme.2011.03.001
Recommended Citation
Muralidhar, Akshay; Borbon, Ivan A; Esharif, Dyadin M; Ke, Wangjing; Manacheril, Rinu; Daines, Michael; and Erickson, Robert P, "Pulmonary function and pathology in hydroxypropyl-beta-cyclodextin-treated and untreated Npc1⁻/⁻ mice." (2011). SJHMC Faculty. 17.
https://scholar.barrowneuro.org/sjhmc/17