Pulmonary function and pathology in hydroxypropyl-beta-cyclodextin-treated and untreated Npc1-/- mice

Akshay Muralidhar, Ivan A. Borbon, Dyadin M. Esharif, Wangjing Ke, Rinu Manacheril, Michael Daines, Robert P. Erickson

Research output: Contribution to journalArticlepeer-review

24 Scopus citations


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 Npc1NIH/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.

Original languageEnglish (US)
Pages (from-to)142-147
Number of pages6
JournalMolecular Genetics and Metabolism
Issue number2
StatePublished - Jun 2011


  • Foamy macrophages
  • Hydroxypropyl-beta-cyclodextrins
  • Intracellular cholesterol transport
  • Niemann-Pick C1
  • Small animal ventilator

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Molecular Biology
  • Genetics
  • Endocrinology


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