Search Results - "Bercury, S.D."
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Intracerebroventricular delivery of glucocerebrosidase reduces substrates and increases lifespan in a mouse model of neuronopathic Gaucher disease
Published in Experimental neurology (01-10-2010)“…Gaucher disease is caused by a deficit in the enzyme glucocerebrosidase. As a consequence, degradation of the glycolipids glucosylceramide (GluCer) and…”
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Journal Article -
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33. Systemic Delivery of AAV8/DC190-GAA to Pompe Mice Normalizes Muscle Pathology and Corrects Motor Function Deficits
Published in Molecular therapy (01-05-2006)“…Pompe disease, a lysosomal storage disorder, is generally known for it's characteristic degenerative myopathy. Massive accumulation of glycogen in lysosomes of…”
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Journal Article