A pilot study of direct delivery of hydroxypropyl-beta-cyclodextrin to the lung by the nasal route in a mouse model of Niemann-Pick C1 disease: motor performance is unaltered and lung disease is worsened

Shortened primary cilium length and dysregulated Sonic hedgehog signaling in Niemann-Pick C1 disease

(2017) Sonia Canterini et al.   HUMAN MOLECULAR GENETICS

Extensive macrophage accumulation in young and old Niemann–Pick C1 model mice involves the alternative, M2, activation pathway and inhibition of macrophage apoptosis

(2016) Gail Deutsch et al.   GENE

Pulmonary macrophage transplantation therapy

(2014) Takuji Suzuki et al.   NATURE

A marked paucity of granule cells in the developing cerebellum of the Npc1−/− mouse is corrected by a single injection of hydroxypropyl-β-cyclodextrin

(2014) S. Nusca et al.   NEUROBIOLOGY OF DISEASE

Current controversies in Niemann–Pick C1 disease: steroids or gangliosides; neurons or neurons and glia

(2013) Robert P. Erickson   JOURNAL OF APPLIED GENETICS

Barrier Mechanisms in the Developing Brain

(2012) Norman R. Saunders et al.   Frontiers in Pharmacology

A novel mouse model of Niemann–Pick type C disease carrying a D1005G-Npc1 mutation comparable to commonly observed human mutations

(2011) Robert A. Maue et al.   HUMAN MOLECULAR GENETICS

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

(2011) Akshay Muralidhar et al.   MOLECULAR GENETICS AND METABOLISM

Increase in excitability of RPeD11 results in memory enhancement of juvenile and adult Lymnaea stagnalis by predator-induced stress

(2010) Hiroshi Sunada et al.   NEUROBIOLOGY OF LEARNING AND MEMORY

Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid

(2009) Benny Liu et al.   JOURNAL OF LIPID RESEARCH

Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression

(2009) Cristin D. Davidson et al.   PLoS One

Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1-/- mouse

(2009) B. Liu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Astrocyte-only Npc1 reduces neuronal cholesterol and triples life span ofNpc1-/-mice

(2008) Min Zhang et al.   JOURNAL OF NEUROSCIENCE RESEARCH