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Updated: Jun 3, 2026
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Phenotyping Mouse Pulmonary Function In Vivo with the Lung Diffusing Capacity
Published on: January 6, 2015
Pulmonary function and pathology in hydroxypropyl-beta-cyclodextin-treated and untreated Npc1⁻/⁻ mice
Akshay Muralidhar1, Ivan A Borbon, Dyadin M Esharif
1Dept of Pediatrics, University of Arizona, Tucson, AZ 85724, USA.
Molecular Genetics and Metabolism
|April 5, 2011
Summary
Niemann-Pick C1 (NPC1) disease causes lung dysfunction, characterized by foamy macrophages and increased lung weight. Cholesterol and collagen also rise, with limited improvement from HPBCD treatment in mouse models.
Area of Science:
- Pulmonary Medicine
- Neurodegenerative Disorders
- Biochemistry
Background:
- Lung dysfunction is a key feature of Niemann-Pick C1 (NPC1), a neurodegenerative disorder.
- The Npc1(NIH/NIH) mouse model exhibits specific pulmonary pathologies relevant to NPC1 disease.
Purpose of the Study:
- To investigate the pulmonary disease characteristics in the Npc1(NIH/NIH) mouse model.
- To assess the impact of hydroxypropyl-β-cyclodextrin (HPBCD) treatment on lung pathology in NPC1 mice.
Main Methods:
- Histological examination of lung tissue to identify cellular changes.
- Pulmonary function testing using a flexiVent small animal ventilator.
- Biochemical analysis of lung cholesterol and collagen levels.
Main Results:
- Histology revealed abundant alveolar foamy macrophages but no alveolar proteinosis.
- Significant increases in lung weight, inspiratory capacity, elastance, and hysterisivity were observed.
- Lung cholesterol and collagen levels were markedly elevated in Npc1(-/-) mice.
Conclusions:
- The Npc1(NIH/NIH) mouse model displays distinct lung pathology, including foamy macrophages and altered mechanics.
- HPBCD treatment showed limited efficacy in ameliorating these pulmonary parameters, even with extended lifespan.
- These findings highlight the specific challenges in treating lung dysfunction in Niemann-Pick C1 disease.
