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


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..

View full abstract


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