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B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study

Molecular therapy. Methods & clinical development, 2014-01, Vol.1 (C), p.14033-14033, Article 14033

2014

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Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heart

Circulation (New York, N.Y.), 2002, Vol.105 (1), p.93-98

2002

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Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease

Human gene therapy, 2016-01, Vol.27 (1), p.43-59

2016

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Pompe disease diagnosis and management guideline

Genetics in medicine, 2006-05, Vol.8 (5), p.267-288

2006

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Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy

The EMBO journal, 2000-09, Vol.19 (17), p.4439-4448

2000

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Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization

Nature medicine, 2002-06, Vol.8 (6), p.607-612

2002

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Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

Gene therapy, 1999-06, Vol.6 (6), p.973-985

1999

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BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease

Clinical immunology (Orlando, Fla.), 2015-06, Vol.158 (2), p.140-147

2015

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Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent

PloS one, 2014-06, Vol.9 (6), p.e98336

2014

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Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

Methods (San Diego, Calif.), 2002-10, Vol.28 (2), p.158-167

2002

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Targeted approaches to induce immune tolerance for Pompe disease therapy

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.15053-15053, Article 15053

2016

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Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults

Human gene therapy, 2004-01, Vol.15 (1), p.93-128

2004

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Successful Production of Pseudotyped rAAV Vectors Using a Modified Baculovirus Expression System

Molecular therapy, 2005-12, Vol.12 (6), p.1217-1225

2005

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Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy

Molecular therapy, 2002-07, Vol.6 (1), p.106-112

2002

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Gene Delivery to Skeletal Muscle Results in Sustained Expression and Systemic Delivery of a Therapeutic Protein

Proceedings of the National Academy of Sciences - PNAS, 1996-11, Vol.93 (24), p.14082-14087

1996

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Sustained Secretion of Human Alpha-1-Antitrypsin from Murine Muscle Transduced with Adeno-Associated Virus Vectors

Proceedings of the National Academy of Sciences - PNAS, 1998-11, Vol.95 (24), p.14384-14388

1998

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CMV-β-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1α promoter and results in therapeutic levels of human factor X in mice

Human gene therapy, 2001-03, Vol.12 (5), p.563-573

2001

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Virus-based gene delivery systems

Clinical pharmacokinetics, 2002, Vol.41 (12), p.901-911

2002

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Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

Gene therapy, 2005-09, Vol.12 (18), p.1405-1409

2005

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In vivo murine left ventricular pressure-volume relations by miniaturized conductance micromanometry

American journal of physiology. Heart and circulatory physiology, 1998-04, Vol.274 (4), p.H1416-H1422

1998

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Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid α-glucosidase in a model of glycogen storage disease type II

Human gene therapy, 2005, Vol.16 (1), p.68-80

2005

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Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease

Development (Cambridge), 2004-06, Vol.131 (12), p.3007-3019

2004

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Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer

Gene therapy, 2006-02, Vol.13 (4), p.321-329

2006

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Correction of the Enzymatic and Functional Deficits in a Model of Pompe Disease Using Adeno-associated Virus Vectors

Molecular therapy, 2002-05, Vol.5 (5), p.571-578

2002

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Myoblast cell grafting into heart muscle: cellular biology and potential applications

Annual review of physiology, 1999-01, Vol.61 (1), p.219-242

1999

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B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study

Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heart

Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease

Pompe disease diagnosis and management guideline

Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy

Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization

Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease

Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent

Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors

Targeted approaches to induce immune tolerance for Pompe disease therapy

Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults

Successful Production of Pseudotyped rAAV Vectors Using a Modified Baculovirus Expression System

Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy

Gene Delivery to Skeletal Muscle Results in Sustained Expression and Systemic Delivery of a Therapeutic Protein

Sustained Secretion of Human Alpha-1-Antitrypsin from Murine Muscle Transduced with Adeno-Associated Virus Vectors

CMV-β-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1α promoter and results in therapeutic levels of human factor X in mice

Virus-based gene delivery systems

Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

In vivo murine left ventricular pressure-volume relations by miniaturized conductance micromanometry

Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid α-glucosidase in a model of glycogen storage disease type II

Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease

Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer

Correction of the Enzymatic and Functional Deficits in a Model of Pompe Disease Using Adeno-associated Virus Vectors

Myoblast cell grafting into heart muscle: cellular biology and potential applications

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