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Abnormal development of NG2+PDGFR-α+ neural progenitor cells leads to neonatal hydrocephalus in a ciliopathy mouse model
Journal article   Peer reviewed

Abnormal development of NG2+PDGFR-α+ neural progenitor cells leads to neonatal hydrocephalus in a ciliopathy mouse model

Calvin S Carter, Timothy W Vogel, Qihong Zhang, Seongjin Seo, Ruth E Swiderski, Thomas O Moninger, Martin D Cassell, Daniel R Thedens, Kim M Keppler-Noreuil, Peggy Nopoulos, …
Nature medicine, Vol.18(12), pp.1797-1804
12/2012
DOI: 10.1038/nm.2996
PMCID: PMC3684048
PMID: 23160237

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Abstract

Hydrocephalus is a common neurological disorder that leads to expansion of the cerebral ventricles and is associated with a high rate of morbidity and mortality. Most neonatal cases are of unknown etiology and are likely to have complex inheritance involving multiple genes and environmental factors. Identifying molecular mechanisms for neonatal hydrocephalus and developing noninvasive treatment modalities are high priorities. Here we use a hydrocephalic mouse model of the human ciliopathy Bardet-Biedl Syndrome (BBS) and identify a role for neural progenitors in the pathogenesis of neonatal hydrocephalus. We found that hydrocephalus in this mouse model is caused by aberrant platelet-derived growth factor receptor α (PDGFR-α) signaling, resulting in increased apoptosis and impaired proliferation of chondroitin sulfate proteoglycan 4 (also known as neuron-glial antigen 2 or NG2)(+)PDGFR-α(+) neural progenitors. Targeting this pathway with lithium treatment rescued NG2(+)PDGFR-α(+) progenitor cell proliferation in BBS mutant mice, reducing their ventricular volume. Our findings demonstrate that neural progenitors are crucial in the pathogenesis of neonatal hydrocephalus, and we identify new therapeutic targets for this common neurological disorder.
Immunohistochemistry In Situ Nick-End Labeling Immunoprecipitation Receptor, Platelet-Derived Growth Factor alpha - metabolism Lithium - pharmacology Proteoglycans - metabolism Bromodeoxyuridine Hydrocephalus - etiology Male Antigens - metabolism DNA Primers - genetics Neural Stem Cells - cytology Blotting, Western Magnetic Resonance Imaging Animals Mice, Mutant Strains Bardet-Biedl Syndrome - pathology Female Signal Transduction - physiology Cell Proliferation - drug effects Mice Apoptosis - physiology Neural Stem Cells - metabolism Real-Time Polymerase Chain Reaction

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