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Helen Beard

Boston Children's Hospital

17

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385

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AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains

2020 Molecular therapy. Methods & clinical development
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Is the eye a window to the brain in Sanfilippo syndrome

2020 Acta neuropathologica communications
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A novel conditional Sgsh knockout mouse model recapitulates phenotypic and neuropathic deficits of Sanfilippo syndrome

2017 Journal of Inherited Metabolic Disease
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Axonal dystrophy in the brain of mice with Sanfilippo syndrome

2017 Experimental Neurology
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A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome

2016 Human Gene Therapy
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Continual Low-Dose Infusion of Sulfamidase Is Superior to Intermittent High-Dose Delivery in Ameliorating Neuropathology in the MPS IIIA Mouse Brain

2015
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Delivery of therapeutic protein for prevention of neurodegenerative changes: comparison of different CSF-delivery methods.

2015 Experimental Neurology
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Disease stage determines the efficacy of treatment of a paediatric neurodegenerative disease

2014 European Journal of Neuroscience
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Helper-dependent canine adenovirus vector-mediated transgene expression in a neurodegenerative lysosomal storage disorder.

2012 Gene
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Enzyme replacement reduces neuropathology in MPS IIIA dogs

2011 Neurobiology of Disease
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Citations (43)