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Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I

Human gene therapy, 2017-07, Vol.28 (7), p.576-587

2017

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Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer

Human gene therapy, 2017-08, Vol.28 (8), p.626-638

2017

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Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

Frontiers in molecular neuroscience, 2021-05, Vol.14, p.618360-618360

2021

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Definition of a novel growth factor-dependent signal cascade for the suppression of bile acid biosynthesis

Genes & development, 2003-07, Vol.17 (13), p.1581-1591

2003

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Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome

Molecular genetics and metabolism reports, 2023-03, Vol.34, p.100956-100956, Article 100956

2023

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Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

Molecular genetics and metabolism reports, 2020-09, Vol.24, p.100604-100604, Article 100604

2020

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Gene transfer and hepatic overexpression of the HDL receptor SR-BI reduces atherosclerosis in the cholesterol-fed LDL receptor-deficient mouse

Arteriosclerosis, thrombosis, and vascular biology, 2000-03, Vol.20 (3), p.721-727

2000

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Overexpression of the HDL receptor SR-BI alters plasma HDL and bile cholesterol levels

Nature (London), 1997-05, Vol.387 (6631), p.414-417

1997

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The lipidation status of acute-phase protein serum amyloid A determines cholesterol mobilization via scavenger receptor class B, type I

Biochemical journal, 2007-02, Vol.402 (1), p.117-124

2007

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Fibrates Down-regulate Hepatic Scavenger Receptor Class B Type I Protein Expression in Mice

The Journal of biological chemistry, 2003-03, Vol.278 (10), p.7884-7890

2003

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Hypochlorite-modified High Density Lipoprotein, a High Affinity Ligand to Scavenger Receptor Class B, Type I, Impairs High Density Lipoprotein-dependent Selective Lipid Uptake and Reverse Cholesterol Transport

The Journal of biological chemistry, 2002-08, Vol.277 (35), p.32172-32179

2002

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Overexpression of SR-BI by Adenoviral Vector Reverses the Fibrateinduced Hypercholesterolemia of Apolipoprotein E-deficient Mice

The Journal of biological chemistry, 2003-12, Vol.278 (52), p.52559-52563

2003

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A model system for in vivo gene transfer into the central nervous system using an adenoviral vector

Nature genetics, 1993-03, Vol.3 (3), p.219-223

1993

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Transcytosis of Lipoprotein Lipase across Cultured Endothelial Cells Requires Both Heparan Sulfate Proteoglycans and the Very Low Density Lipoprotein Receptor

The Journal of biological chemistry, 2001-03, Vol.276 (12), p.8934-8941

2001

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Scavenger receptor class B, type I on non-malignant and malignant human epithelial cells mediates cholesteryl ester-uptake from high density lipoproteins

The international journal of biochemistry & cell biology, 2003-04, Vol.35 (4), p.441-454

2003

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The Very Low Density Lipoprotein Receptor Mediates the Cellular Catabolism of Lipoprotein Lipase and Urokinase-Plasminogen Activator Inhibitor Type I Complexes (∗)

The Journal of biological chemistry, 1995-11, Vol.270 (44), p.26550-26557

1995

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A tetracycline-regulated adenoviral expression system for in vivo delivery of transgenes to lung and liver

The journal of gene medicine, 2003-07, Vol.5 (7), p.567-575

2003

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Gene therapy for cardiovascular disease

Current Opinion in Pharmacology, 2001-04, Vol.1 (2), p.197-202

Kozarsky, Karen F

2001

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Hepatic expression of the catalytic subunit of the apolipoprotein B mRNA editing enzyme (apobec-1) ameliorates hypercholesterolemia in LDL receptor-deficient rabbits

Human gene therapy, 1996-05, Vol.7 (8), p.943-957

1996

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Gene therapy: adenovirus vectors

Current opinion in genetics & development, 1993-06, Vol.3 (3), p.499-503

1993

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Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene

Nature genetics, 1996-05, Vol.13 (1), p.54-62

1996

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In vivo correction of low density lipoprotein receptor deficiency in the Watanabe heritable hyperlipidemic rabbit with recombinant adenoviruses

The Journal of biological chemistry, 1994-05, Vol.269 (18), p.13695-13702

1994

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Restoration of LDL Receptor Activity in Mutant Cells by Intercellular Junctional Communication

Science (American Association for the Advancement of Science), 1987-01, Vol.235 (4784), p.69-73

1987

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The atherogenic lipoprotein Lp(a) is internalized and degraded in a process mediated by the VLDL receptor

The Journal of clinical investigation, 1997-11, Vol.100 (9), p.2170-2181

1997

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Localization of the steroidogenic acute regulatory protein in human tissues

The journal of clinical endocrinology and metabolism, 1997-12, Vol.82 (12), p.4243-4251

1997

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Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I

Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System-Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer

Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

Definition of a novel growth factor-dependent signal cascade for the suppression of bile acid biosynthesis

Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome

Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

Gene transfer and hepatic overexpression of the HDL receptor SR-BI reduces atherosclerosis in the cholesterol-fed LDL receptor-deficient mouse

Overexpression of the HDL receptor SR-BI alters plasma HDL and bile cholesterol levels

The lipidation status of acute-phase protein serum amyloid A determines cholesterol mobilization via scavenger receptor class B, type I

Fibrates Down-regulate Hepatic Scavenger Receptor Class B Type I Protein Expression in Mice

Hypochlorite-modified High Density Lipoprotein, a High Affinity Ligand to Scavenger Receptor Class B, Type I, Impairs High Density Lipoprotein-dependent Selective Lipid Uptake and Reverse Cholesterol Transport

Overexpression of SR-BI by Adenoviral Vector Reverses the Fibrateinduced Hypercholesterolemia of Apolipoprotein E-deficient Mice

A model system for in vivo gene transfer into the central nervous system using an adenoviral vector

Transcytosis of Lipoprotein Lipase across Cultured Endothelial Cells Requires Both Heparan Sulfate Proteoglycans and the Very Low Density Lipoprotein Receptor

Scavenger receptor class B, type I on non-malignant and malignant human epithelial cells mediates cholesteryl ester-uptake from high density lipoproteins

The Very Low Density Lipoprotein Receptor Mediates the Cellular Catabolism of Lipoprotein Lipase and Urokinase-Plasminogen Activator Inhibitor Type I Complexes (∗)

A tetracycline-regulated adenoviral expression system for in vivo delivery of transgenes to lung and liver

Gene therapy for cardiovascular disease

Hepatic expression of the catalytic subunit of the apolipoprotein B mRNA editing enzyme (apobec-1) ameliorates hypercholesterolemia in LDL receptor-deficient rabbits

Gene therapy: adenovirus vectors

Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene

In vivo correction of low density lipoprotein receptor deficiency in the Watanabe heritable hyperlipidemic rabbit with recombinant adenoviruses

Restoration of LDL Receptor Activity in Mutant Cells by Intercellular Junctional Communication

The atherogenic lipoprotein Lp(a) is internalized and degraded in a process mediated by the VLDL receptor

Localization of the steroidogenic acute regulatory protein in human tissues

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