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The tandem mass spectrometry newborn screening experience in North Carolina: 1997–2005

Journal of inherited metabolic disease, 2006-02, Vol.29 (1), p.76-85

2006

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Immunomodulatory, liver depot gene therapy for Pompe disease

Cellular immunology, 2019-08, Vol.342, p.103737-103737, Article 103737

2019

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A comprehensive testing algorithm for the diagnosis of Fabry disease in males and females

Molecular genetics and metabolism, 2020-07, Vol.130 (3), p.209-214

2020

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Bezafibrate induces autophagy and improves hepatic lipid metabolism in glycogen storage disease type Ia

Human molecular genetics, 2019-01, Vol.28 (1), p.143-154

2019

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Successful AAV8 readministration: Suppression of capsid‐specific neutralizing antibodies by a combination treatment of bortezomib and CD20 mAb in a mouse model of Pompe disease

The journal of gene medicine, 2023-08, Vol.25 (8), p.e3509-n/a

2023

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Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance

American journal of human genetics, 2007-11, Vol.81 (5), p.1042-1049

2007

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Preclinical Development of New Therapy for Glycogen Storage Diseases

Current gene therapy, 2015-01, Vol.15 (4), p.338

2015

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Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease

Human gene therapy, 2012-05, Vol.23 (5), p.460

2012

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The Role of Hepatocyte Hemojuvelin in the Regulation of Bone Morphogenic Protein-6 and Hepcidin Expression in Vivo

The Journal of biological chemistry, 2010-05, Vol.285 (22), p.16416-16423

2010

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Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2 8 vector-mediated gene therapy in murine phenylketonuria

Gene therapy, 2006-03, Vol.13 (5), p.457-462

2006

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Gene therapy for glycogen storage diseases

Human molecular genetics, 2019-10, Vol.28 (R1), p.R31-R41

2019

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Itavastatin and resveratrol increase triosephosphate isomerase protein in a newly identified variant of TPI deficiency

Disease models & mechanisms, 2022-05, Vol.15 (5)

2022

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Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia

Molecular therapy. Methods & clinical development, 2023-06, Vol.29, p.108-119

2023

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Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice

Blood, 2010-04, Vol.115 (16), p.3374-3381

2010

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Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

Molecular genetics and metabolism, 2011-06, Vol.103 (2), p.107-112

2011

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Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease

Molecular therapy, 2010-02, Vol.18 (2), p.353-360

2010

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Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy

JIMD reports, 2023-09, Vol.64 (5), p.393-400

2023

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Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease

Communications biology, 2021-05, Vol.4 (1), p.524-524, Article 524

2021

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A beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.114-119

2016

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Elevation of guanidinoacetate in newborn dried blood spots and impact of early treatment in GAMT deficiency

Molecular genetics and metabolism, 2013-06, Vol.109 (2), p.215-217

2013

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Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease

Gene therapy, 2010-12, Vol.17 (12), p.1500-1505

2010

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Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease

Molecular therapy. Methods & clinical development, 2019-12, Vol.15, p.383-391

2019

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Emerging therapies for glycogen storage disease type I

Trends in endocrinology and metabolism, 2009-07, Vol.20 (5), p.252-258

2009

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Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia

Gene therapy, 2006-09, Vol.13 (17), p.1281-1289

2006

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Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.133-142

2020

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The tandem mass spectrometry newborn screening experience in North Carolina: 1997–2005

Immunomodulatory, liver depot gene therapy for Pompe disease

A comprehensive testing algorithm for the diagnosis of Fabry disease in males and females

Bezafibrate induces autophagy and improves hepatic lipid metabolism in glycogen storage disease type Ia

Successful AAV8 readministration: Suppression of capsid‐specific neutralizing antibodies by a combination treatment of bortezomib and CD20 mAb in a mouse model of Pompe disease

Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance

Preclinical Development of New Therapy for Glycogen Storage Diseases

Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease

The Role of Hepatocyte Hemojuvelin in the Regulation of Bone Morphogenic Protein-6 and Hepcidin Expression in Vivo

Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2 8 vector-mediated gene therapy in murine phenylketonuria

Gene therapy for glycogen storage diseases

Itavastatin and resveratrol increase triosephosphate isomerase protein in a newly identified variant of TPI deficiency

Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia

Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice

Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease

Screening data from 19 patients with late‐onset Pompe disease for a phase I clinical trial of AAV8 vector‐mediated gene therapy

Three-dimensional tissue-engineered human skeletal muscle model of Pompe disease

A beta-blocker, propranolol, decreases the efficacy from enzyme replacement therapy in Pompe disease

Elevation of guanidinoacetate in newborn dried blood spots and impact of early treatment in GAMT deficiency

Hydrostatic isolated limb perfusion with adeno-associated virus vectors enhances correction of skeletal muscle in Pompe disease

Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease

Emerging therapies for glycogen storage disease type I

Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia

Comparisons of Infant and Adult Mice Reveal Age Effects for Liver Depot Gene Therapy in Pompe Disease

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