Search Results - "Bagel, J H"
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Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain
Published in Gene therapy (01-01-2014)“…Canine models have many advantages for evaluating therapy of human central nervous system (CNS) diseases. In contrast to nonhuman primate models, naturally…”
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Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease
Published in Science translational medicine (25-02-2015)“…Niemann-Pick type C1 (NPC) disease is a lysosomal storage disease caused by mutations in the NPC1 gene, leading to an increase in unesterified cholesterol and…”
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Cholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann-Pick C1 (NPC1)
Published in Human molecular genetics (15-11-2014)“…Niemann-Pick C1 (NPC1) disease is a rare, neurodegenerative lysosomal cholesterol storage disorder, typified by progressive cognitive and motor function…”
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