Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery von Fu, Haiyan, Cataldi, Marcela P, Ware, Tierra A, Zaraspe, Kimberly, Meadows, Aaron S, Murrey, Darren A, McCarty, Douglas M

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Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates von Murrey, Darren A, Naughton, Bartholomew J, Duncan, F Jason, Meadows, Aaron S, Ware, Tierra A, Campbell, Katie J, Bremer, William G, Walker, Christopher M, Goodchild, Laurie, Bolon, Brad, La Perle, Krista, Flanigan, Kevin M, McBride, Kim L, McCarty, Douglas M, Fu, Haiyan

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Feasibility and Safety of Systemic rAAV9-h NAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates von Murrey, Darren A., Naughton, Bartholomew J., Duncan, F. Jason, Meadows, Aaron S., Ware, Tierra A., Campbell, Katie J., Bremer, William G., Walker, Christopher M., Goodchild, Laurie, Bolon, Brad, La Perle, Krista, Flanigan, Kevin M., McBride, Kim L., McCarty, Douglas M., Fu, Haiyan

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Amyloidosis, synucleinopathy, and prion encephalopathy in a neuropathic lysosomal storage disease: the CNS-biomarker potential of peripheral blood von Naughton, Bartholomew J, Duncan, F Jason, Murrey, Darren, Ware, Tierra, Meadows, Aaron, McCarty, Douglas M, Fu, Haiyan

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Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery von Fu, Haiyan, Meadows, Aaron S., Ware, Tierra, Mohney, Robert P., McCarty, Douglas M.

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Functional correction and reversal of MPS IIIA in a mouse model by an intravenous hSGSH gene delivery using a self-complementary AAV9 vector von McCarty, Douglas M., Zaraspe, Kimberly, Murrey, Darren, Ware, Tierra, Fu, Haiyan

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Correction of broad molecular impairments in a mouse model of MPS IIIA by systemic rAAVrh74-hSGSH gene delivery von Fu, Haiyan, Duncan, F. Jason, Naughton, Bartholomew, Zaraspe, Kimberly, Murrey, Darren, Ware, Tierra, Meadows, Aaron, McCarty, Douglas M.

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Amyloidosis, Synucleinopathy, and Prion Encephalopathy in a Neuropathic Lysosomal Storage Disease: The CNS-Biomarker Potential of Peripheral Blood: e80142 von Naughton, Bartholomew J, Duncan, F Jason, Murrey, Darren, Ware, Tierra, Meadows, Aaron, McCarty, Douglas M, Fu, Haiyan

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Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating MPS IIIB: Toxicology, Bio-distribution and Immunological Assessments in Primates von Murrey, Darren A, Naughton, Bartholomew J, Duncan, F Jason, Meadows, Aaron S, Ware, Tierra Amber, Campbell, Katie, Bremer, William G, Walker, Christopher, Goodchild, Laurie, Bolon, Brad, La Perle, Krista, Flanigan, Kevin, McBride, Kim L, McCarty, Douglas M, Fu, Haiyan

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