Search Results - "Naughton, Bartholomew J"
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Blood genome-wide transcriptional profiles reflect broad molecular impairments and strong blood-brain links in Alzheimer's disease
Published in Journal of Alzheimer's disease (01-01-2015)“…To date, little is known regarding the etiology and disease mechanisms of Alzheimer's disease (AD). There is a general urgency for novel approaches to advance…”
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Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice
Published in Molecular therapy (01-04-2015)“…Mucopolysaccharidosis (MPS) IIIA is a neuropathic lysosomal storage disease caused by deficiency in N-sulfoglucosamine sulfohydrolase (SGSH). Genome-wide gene…”
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Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates
Published in Human gene therapy. Clinical development (01-06-2014)“…No treatment is currently available for mucopolysaccharidosis (MPS) IIIB, a neuropathic lysosomal storage disease caused by autosomal recessive defect in…”
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Amyloidosis, synucleinopathy, and prion encephalopathy in a neuropathic lysosomal storage disease: the CNS-biomarker potential of peripheral blood
Published in PloS one (21-11-2013)“…Mucopolysaccharidosis (MPS) IIIB is a devastating neuropathic lysosomal storage disease with complex pathology. This study identifies molecular signatures in…”
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5
Dopamine transporter inhibition is necessary for cocaine-induced increases in dendritic spine density in the nucleus accumbens
Published in Synapse (New York, N.Y.) (01-06-2011)“…Repeated exposure to cocaine produces changes in the nervous system that facilitate drug‐seeking behaviors. These drug‐seeking behaviors have been studied with…”
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A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB
Published in Human gene therapy. Clinical development (01-12-2015)“…No treatment is currently available for mucopolysaccharidosis (MPS) IIIB, a neuropathic lysosomal storage disease due to defect in α-N-acetylglucosaminidase…”
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Amyloidosis, Synucleinopathy, and Prion Encephalopathy in a Neuropathic Lysosomal Storage Disease: The CNS-Biomarker Potential of Peripheral Blood: e80142
Published in PloS one (01-11-2013)“…Mucopolysaccharidosis (MPS) IIIB is a devastating neuropathic lysosomal storage disease with complex pathology. This study identifies molecular signatures in…”
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Journal Article -
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Brain Region and Cell Type Specific Approaches to Study Drug Abuse
Published 01-01-2011“…Drug dependence is a persistent problem throughout the world. Once addicted to a drug, many users have difficulty quitting use, even when they desire to stop…”
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Dissertation