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

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..