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Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Proceedings of the National Academy of Sciences - PNAS, 2009-09, Vol.106 (38), p.16363-16368

2009

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Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes

Human gene therapy, 2013-06, Vol.24 (6), p.630

2013

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Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo

Circulation research, 2006-08, Vol.99 (4), p.e3-e9

2006

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Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

Human gene therapy. Clinical development, 2017-12, Vol.28 (4), p.208

2017

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The Pharmacology of Gene and Cell Therapy

Molecular therapy. Methods & clinical development, 2018-03, Vol.8 (C), p.181-182

2018

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Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

Human gene therapy. Clinical development, 2015-09, Vol.26 (3), p.185

2015

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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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Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites

Human gene therapy, 2007-03, Vol.18 (3), p.245

2007

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Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy

Science translational medicine, 2023-01, Vol.15 (677), p.eabo1815

2023

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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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Functional Consequences of PDK4 Deficiency in Doberman Pinscher Fibroblasts

Scientific reports, 2020-03, Vol.10 (1), p.3930-3930, Article 3930

2020

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Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

The New England journal of medicine, 2009-08, Vol.361 (7), p.725-727

2009

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The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina

Human gene therapy, 2012-10, Vol.23 (10), p.1101

2012

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Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa

Human gene therapy, 2015-09, Vol.26 (9), p.593

2015

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LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile

Investigative ophthalmology & visual science, 2014-12, Vol.55 (12), p.7739-7753

2014

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Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia

Journal of inherited metabolic disease, 2018-12, Vol.41 (6), p.977-984

2018

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Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease

Molecular therapy, 2010-03, Vol.18 (3), p.502-510

2010

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Pompe disease gene therapy: Gene Therapy

Human molecular genetics, 2011-04, Vol.20 (1), p.R61-R68

2011

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Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection

Molecular therapy, 2006-06, Vol.13 (6), p.1074-1084

2006

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In vitro evaluation of mitochondrial dysfunction and treatment with adeno-associated virus vector in fibroblasts from Doberman Pinschers with dilated cardiomyopathy and a pyruvate dehydrogenase kinase 4 mutation

American journal of veterinary research, 2016-02, Vol.77 (2), p.156-161

2016

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A Preclinical Study in Rhesus Macaques for Cystic Fibrosis to Assess Gene Transfer and Transduction by AAV1 and AAV5 with a Dual-Luciferase Reporter System

Human gene therapy. Clinical development, 2017-09, Vol.28 (3), p.145

2017

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Regression of schwannomas induced by adeno-associated virus-mediated delivery of caspase-1

Human gene therapy, 2013-02, Vol.24 (2), p.152

2013

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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years

Archives of ophthalmology (1960), 2012-01, Vol.130 (1), p.9

2012

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Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis

Human gene therapy, 2006-08, Vol.17 (8), p.845

2006

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Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa

Human gene therapy. Clinical development, 2013-03, Vol.24 (1), p.23

2013

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Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes

Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

The Pharmacology of Gene and Cell Therapy

Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

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

Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites

Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy

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

Functional Consequences of PDK4 Deficiency in Doberman Pinscher Fibroblasts

Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina

Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa

LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile

Long-term safety and efficacy of AAV gene therapy in the canine model of glycogen storage disease type Ia

Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease

Pompe disease gene therapy: Gene Therapy

Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection

In vitro evaluation of mitochondrial dysfunction and treatment with adeno-associated virus vector in fibroblasts from Doberman Pinschers with dilated cardiomyopathy and a pyruvate dehydrogenase kinase 4 mutation

A Preclinical Study in Rhesus Macaques for Cystic Fibrosis to Assess Gene Transfer and Transduction by AAV1 and AAV5 with a Dual-Luciferase Reporter System

Regression of schwannomas induced by adeno-associated virus-mediated delivery of caspase-1

Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years

Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis

Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa

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