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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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Chlamydia trachomatis IgG3 seropositivity is associated with gastroschisis

Journal of perinatology, 2015-11, Vol.35 (11), p.930-934

2015

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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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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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Outcomes of a second non-myeloablative allogeneic stem cell transplantation following graft rejection

Bone marrow transplantation (Basingstoke), 2008-01, Vol.41 (1), p.39-43

2008

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High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 rep and cap

Gene therapy, 1999-06, Vol.6 (6), p.986-993

1999

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Advancements in AAV-mediated Gene Therapy for Pompe Disease

Journal of neuromuscular diseases, 2020-01, Vol.7 (1), p.15-31

2020

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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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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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Transcriptome assessment of the Pompe (Gaa-/-) mouse spinal cord indicates widespread neuropathology

Physiological genomics, 2016-11, Vol.48 (11), p.785-794

2016

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Ventricular arrhythmia in the X-linked cardiomyopathy Barth syndrome

Pediatric cardiology, 2005-10, Vol.26 (5), p.632-637

2005

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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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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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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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Oxygen saturation as a screening test for critical congenital heart disease: a preliminary study

Pediatric cardiology, 2002-07, Vol.23 (4), p.403-409

2002

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Recombinant adeno-associated virus type 2 replication and packaging is entirely supported by a herpes simplex virus type 1 amplicon expressing Rep and Cap

Journal of Virology, 1997-11, Vol.71 (11), p.8780-8789

1997

Volltextzugriff (PDF)

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

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

2006

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Intercellular transfer of the virally derived precursor form of acid α-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy pompe disease

Human gene therapy, 2001-03, Vol.12 (5), p.527-538

2001

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Complete correction of acid α-glucosidase deficiency in pompe disease fibroblasts in vitro, and lysosomally targeted expression in neonatal rat cardiac and skeletal muscle

Gene therapy, 1998-04, Vol.5 (4), p.473-480

1998

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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

Volltextzugriff (PDF)

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

Volltextzugriff (PDF)

Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial

Human gene therapy, 2008-10, Vol.19 (10), p.979

2008

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Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

Human gene therapy, 2009-09, Vol.20 (9), p.999

2009

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Pulse Pressure-Related Changes in Coronary Flow In Vivo Are Modulated by Nitric Oxide and Adenosine

Circulation research, 1996-10, Vol.79 (4), p.849-856

1996

Volltextzugriff (PDF)

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

Chlamydia trachomatis IgG3 seropositivity is associated with gastroschisis

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

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

Outcomes of a second non-myeloablative allogeneic stem cell transplantation following graft rejection

High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 rep and cap

Advancements in AAV-mediated Gene Therapy for Pompe Disease

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

Transcriptome assessment of the Pompe (Gaa-/-) mouse spinal cord indicates widespread neuropathology

Ventricular arrhythmia in the X-linked cardiomyopathy Barth syndrome

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

Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization

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

Oxygen saturation as a screening test for critical congenital heart disease: a preliminary study

Recombinant adeno-associated virus type 2 replication and packaging is entirely supported by a herpes simplex virus type 1 amplicon expressing Rep and Cap

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

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

Intercellular transfer of the virally derived precursor form of acid α-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy pompe disease

Complete correction of acid α-glucosidase deficiency in pompe disease fibroblasts in vitro, and lysosomally targeted expression in neonatal rat cardiac and skeletal muscle

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

Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial

Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

Pulse Pressure-Related Changes in Coronary Flow In Vivo Are Modulated by Nitric Oxide and Adenosine

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