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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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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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Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis

American journal of physiology: Gastrointestinal and liver physiology, 2012-02, Vol.302 (3), p.G296-G308

2012

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Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

Molecular therapy, 2012-06, Vol.20 (6), p.1131-1138

2012

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Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors

Human gene therapy. Clinical development, 2016-12, Vol.27 (4), p.152

2016

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An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery

Genetic vaccines and therapy, 2012-06, Vol.10 (1), p.3-3, Article 3

2012

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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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In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno-associated virus (rAAV) serotypes 2 and 8

The journal of gene medicine, 2007-06, Vol.9 (6), p.462-469

2007

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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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Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse

Investigative ophthalmology & visual science, 2011-09, Vol.52 (10), p.7098-7108

2011

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463. Safety and Biodistribution Study of rAAV2tYF-CB-hRS1 in RS1-deficient Mice

Molecular therapy, 2015-05, Vol.23, p.S184-S184

2015

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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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Biodistribution and toxicology studies of AAV2/rh8-CBA- beta -hexosaminidase after intracranial delivery in C57/BI6 mice

Molecular therapy, 2013-09, Vol.21 (9), p.e27-e28

2013

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Safety and Biodistribution Evaluation of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin, in RS1-Deficient Mice

Human gene therapy. Clinical development, 2015-09, Vol.26 (3), p.177

2015

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Efficient Hepatic Delivery and Expression from a Recombinant Adeno-associated Virus 8 Pseudotyped α1-Antitrypsin Vector

Molecular therapy, 2005-11, Vol.12 (5), p.867-875

2005

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Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

Molecular therapy, 2012-06, Vol.20 (6), p.1287-1287

2012

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Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis

American journal of physiology: Gastrointestinal and liver physiology, 2012, Vol.65 (2)

2012

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Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase

Human gene therapy. Clinical development, 2013-09, Vol.24 (3), p.127

2013

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Next generation treatment of Pompe disease using systemic gene transfer with AAV9

Molecular genetics and metabolism, 2011-02, Vol.102 (2), p.S18-S18

2011

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Reprogramming adipose tissue-derived mesenchymal stem cells into pluripotent stem cells by a mutant adeno-associated viral vector

Human gene therapy. Part B. Methods, 2014-02, Vol.25 (1), p.72

2014

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Systemic correction of a fatty acid oxidation defect by intramuscular injection of a recombinant adeno-associated virus vector

Human gene therapy, 2006-01, Vol.17 (1), p.71

2006

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436. Recombinant Adeno-Associated Virus Gene Therapy for Cob(I)Alamin Adenosyltransferase Deficiency

Molecular therapy, 2004-05, Vol.9 (S1), p.S166-S167

2004

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Systemic Correction of a Fatty Acid Oxidation Defect by Intramuscular Injection of a Recombinant Adeno-Associated Virus Vector

Human gene therapy, 2005-12, p.51214070249001

2005

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Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial

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

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

Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis

Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors

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

An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery

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

In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno-associated virus (rAAV) serotypes 2 and 8

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

Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse

463. Safety and Biodistribution Study of rAAV2tYF-CB-hRS1 in RS1-deficient Mice

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

Biodistribution and toxicology studies of AAV2/rh8-CBA- beta -hexosaminidase after intracranial delivery in C57/BI6 mice

Safety and Biodistribution Evaluation of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin, in RS1-Deficient Mice

Efficient Hepatic Delivery and Expression from a Recombinant Adeno-associated Virus 8 Pseudotyped α1-Antitrypsin Vector

Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis

Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase

Next generation treatment of Pompe disease using systemic gene transfer with AAV9

Reprogramming adipose tissue-derived mesenchymal stem cells into pluripotent stem cells by a mutant adeno-associated viral vector

Systemic correction of a fatty acid oxidation defect by intramuscular injection of a recombinant adeno-associated virus vector

436. Recombinant Adeno-Associated Virus Gene Therapy for Cob(I)Alamin Adenosyltransferase Deficiency

Systemic Correction of a Fatty Acid Oxidation Defect by Intramuscular Injection of a Recombinant Adeno-Associated Virus Vector

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