Journal article

Iron chelation by deferiprone does not rescue the Niemann-Pick Disease Type C1 mouse model

Ya Hui Hung, Amit Lotan, Shlomo Yeshurun, Anna Schroeder, Ashley I Bush

BIOMETALS | SPRINGER | Published : 2020

Abstract

Niemann-Pick Disease Type C (NP-C) is a fatal lysosomal storage disorder with progressive neurodegeneration. In addition to the characteristic cholesterol and lipid overload phenotype, we previously found that altered metal homeostasis is also a pathological feature. Increased brain iron in the Npc1-/- mouse model of NP-C may potentially contribute to neurodegeneration, similar to neurodegenerative diseases such as Alzheimer's and Parkinson's diseases. Deferiprone (DFP) is a brain penetrating iron chelator that has demonstrated effectiveness in preventing neurological deterioration in Parkinson's disease clinical trials. Therefore, we hypothesized that DFP treatment, targeting brain iron ove..

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Grants

Funding Acknowledgements

This work was supported by research grants from the Australian NPC Disease Foundation, Inc.; the Australia-Israel Medical Research Foundation (AUSiMED); Victoria Israel Science Innovation and Technology Scheme Grants for Feasibility and Proof of Concept Projects; Cooperative Research Centre for Mental Health; and the Victorian Government Operational Infrastructure Support Grant