Concepedia

Normalization of Hepatic Homeostasis in the Npc1 Mouse Model of Niemann-Pick Type C Disease Treated with the Histone Deacetylase Inhibitor Vorinostat

Andrew B. Munkacsi, Natalie Hammond, Remy T. Schneider, Dinindu Senanayake, Katsumi Higaki, Kirill Lagutin, Stephen J. Bloor, Daniel Ory, Robert A. Maue, Fannie W. Chen,

Journal of Biological Chemistry · 2016 · 34 citations · 67 references

DOIFull text

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Concepts

Abstract

locus. These results suggest that HDAC inhibitors have utility to treat visceral NP-C disease. However, it is clear that improved blood-brain barrier penetration will be required to alleviate the neurological symptoms of human NP-C disease.

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