Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery
Fu, Haiyan, Cataldi, Marcela P, Ware, Tierra A, Zaraspe, Kimberly, Meadows, Aaron S, Murrey, Darren A, McCarty, Douglas M
Published in Molecular therapy. Methods & clinical development (01.01.2016)
Published in Molecular therapy. Methods & clinical development (01.01.2016)
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Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates
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
Published in Human gene therapy. Clinical development (01.06.2014)
Published in Human gene therapy. Clinical development (01.06.2014)
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Amyloidosis, synucleinopathy, and prion encephalopathy in a neuropathic lysosomal storage disease: the CNS-biomarker potential of peripheral blood
Naughton, Bartholomew J, Duncan, F Jason, Murrey, Darren, Ware, Tierra, Meadows, Aaron, McCarty, Douglas M, Fu, Haiyan
Published in PloS one (21.11.2013)
Published in PloS one (21.11.2013)
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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
Fu, Haiyan, Meadows, Aaron S., Ware, Tierra, Mohney, Robert P., McCarty, Douglas M.
Published in Molecular therapy (01.03.2017)
Published in Molecular therapy (01.03.2017)
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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
McCarty, Douglas M., Zaraspe, Kimberly, Murrey, Darren, Ware, Tierra, Fu, Haiyan
Published in Molecular genetics and metabolism (01.02.2014)
Published in Molecular genetics and metabolism (01.02.2014)
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Correction of broad molecular impairments in a mouse model of MPS IIIA by systemic rAAVrh74-hSGSH gene delivery
Fu, Haiyan, Duncan, F. Jason, Naughton, Bartholomew, Zaraspe, Kimberly, Murrey, Darren, Ware, Tierra, Meadows, Aaron, McCarty, Douglas M.
Published in Molecular genetics and metabolism (01.02.2014)
Published in Molecular genetics and metabolism (01.02.2014)
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Amyloidosis, Synucleinopathy, and Prion Encephalopathy in a Neuropathic Lysosomal Storage Disease: The CNS-Biomarker Potential of Peripheral Blood: e80142
Naughton, Bartholomew J, Duncan, F Jason, Murrey, Darren, Ware, Tierra, Meadows, Aaron, McCarty, Douglas M, Fu, Haiyan
Published in PloS one (01.11.2013)
Published in PloS one (01.11.2013)
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Journal Article
Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating MPS IIIB: Toxicology, Bio-distribution and Immunological Assessments in Primates
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
Published in Human gene therapy. Clinical development (11.03.2014)
Published in Human gene therapy. Clinical development (11.03.2014)
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