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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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Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

Science translational medicine, 2017-11, Vol.9 (418)

2017

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A Liver-Specific Thyromimetic, VK2809, Decreases Hepatosteatosis in Glycogen Storage Disease Type Ia

Thyroid (New York, N.Y.), 2019-08, Vol.29 (8), p.1158-1167

2019

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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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Preclinical Development of New Therapy for Glycogen Storage Diseases

Current gene therapy, 2015-01, Vol.15 (4), p.338-347

2015

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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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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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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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Gene therapy for glycogen storage diseases

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

2019

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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.460-472

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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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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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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Staying on target in gene and cell therapy

Molecular therapy, 2024-09, Vol.32 (9), p.2801-2802

2024

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

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

A Liver-Specific Thyromimetic, VK2809, Decreases Hepatosteatosis in Glycogen Storage Disease Type Ia

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

Preclinical Development of New Therapy for Glycogen Storage Diseases

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

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

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

Gene 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

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

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

Staying on target in gene and cell therapy

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

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

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

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

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