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CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

The Journal of clinical investigation, 2010-04, Vol.120 (4), p.1253-1264

2010

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Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors

Human gene therapy. Part B. Methods, 2015-12, Vol.26 (6), p.228-242

2015

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Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa

Molecular therapy, 2023-07, Vol.31 (7), p.2028-2041

2023

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Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy

Human gene therapy, 2014-07, Vol.25 (7), p.619-630

2014

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Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes

Molecular therapy. Methods & clinical development, 2018-06, Vol.9, p.33-46

2018

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Gene Transfer of Human Acid Sphingomyelinase Corrects Neuropathology and Motor Deficits in a Mouse Model of Niemann-Pick Type A Disease

Proceedings of the National Academy of Sciences - PNAS, 2005-12, Vol.102 (49), p.17822-17827

2005

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The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16006-16006, Article 16006

2016

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In Vivo Potency Testing of Subretinal rAAV5.hCNGB1 Gene Therapy in the Cngb1 Knockout Mouse Model of Retinitis Pigmentosa

Human gene therapy, 2021-10, Vol.32 (19-20), p.1158-1170

2021

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Combination Brain and Systemic Injections of AAV Provide Maximal Functional and Survival Benefits in the Niemann-Pick Mouse

Proceedings of the National Academy of Sciences - PNAS, 2007-05, Vol.104 (22), p.9505-9510

2007

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Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain

Gene therapy, 2018-06, Vol.25 (3), p.205-219

2018

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AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann–Pick A Disease

Molecular therapy, 2005-05, Vol.11 (5), p.754-762

2005

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Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection

Human gene therapy. Part B. Methods, 2017-02, Vol.28 (1), p.23-38

2017

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cAMP activates an ATP-permeable pathway in neonatal rat cardiac myocytes

American Journal of Physiology: Cell Physiology, 2000-07, Vol.279 (1), p.C173-C187

2000

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Electrodiffusional ATP movement through the cystic fibrosis transmembrane conductance regulator

American Journal of Physiology: Cell Physiology, 1998-03, Vol.274 (3), p.C799-C809

1998

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Engineered Capsids for Efficient Gene Delivery to the Retina and Cornea

Human gene therapy, 2020-07, Vol.31 (13-14), p.756-774

2020

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Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis

Cell, 1990-11, Vol.63 (4), p.827-834

1990

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Targeting the urokinase plasminogen activator receptor enhances gene transfer to human airway epithelia

The Journal of clinical investigation, 2000-03, Vol.105 (5), p.589-596

2000

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Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV)

The journal of gene medicine, 2000-11, Vol.2 (6), p.444-454

2000

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Strategies to adapt adenoviral vectors for targeted delivery

Methods in molecular medicine, 2003, Vol.76, p.89

2003

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Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations

iScience, 2021-05, Vol.24 (5), p.102409, Article 102409

2021

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PEGylation of adenovirus with retention of infectivity and protection from neutralizing antibody in vitro and in vivo

Human gene therapy, 1999-05, Vol.10 (8), p.1349-1358

1999

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92. Evaluation of Producer Cell Line Platform for Production of Oversized AAV-FVIII Vectors

Molecular therapy, 2016-05, Vol.24, p.S40-S40

2016

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PEGylated adenovirus for targeted gene therapy

Methods in molecular biology (Clifton, N.J.), 2008, Vol.434, p.133

2008

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Purification and Characterization of Recombinant Cystic Fibrosis Transmembrane Conductance Regulator from Chinese Hamster Ovary and Insect Cells

The Journal of biological chemistry, 1995-07, Vol.270 (28), p.17033-17043

1995

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Modification of an adenoviral vector with biologically selected peptides : A novel strategy for gene delivery to cells of choice

Human gene therapy, 1999-11, Vol.10 (16), p.2615-2626

1999

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CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors

Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa

Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy

Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes

Gene Transfer of Human Acid Sphingomyelinase Corrects Neuropathology and Motor Deficits in a Mouse Model of Niemann-Pick Type A Disease

The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo

In Vivo Potency Testing of Subretinal rAAV5.hCNGB1 Gene Therapy in the Cngb1 Knockout Mouse Model of Retinitis Pigmentosa

Combination Brain and Systemic Injections of AAV Provide Maximal Functional and Survival Benefits in the Niemann-Pick Mouse

Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain

AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann–Pick A Disease

Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection

cAMP activates an ATP-permeable pathway in neonatal rat cardiac myocytes

Electrodiffusional ATP movement through the cystic fibrosis transmembrane conductance regulator

Engineered Capsids for Efficient Gene Delivery to the Retina and Cornea

Defective intracellular transport and processing of CFTR is the molecular basis of most cystic fibrosis

Targeting the urokinase plasminogen activator receptor enhances gene transfer to human airway epithelia

Scaleable chromatographic purification process for recombinant adeno-associated virus (rAAV)

Strategies to adapt adenoviral vectors for targeted delivery

Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations

PEGylation of adenovirus with retention of infectivity and protection from neutralizing antibody in vitro and in vivo

92. Evaluation of Producer Cell Line Platform for Production of Oversized AAV-FVIII Vectors

PEGylated adenovirus for targeted gene therapy

Purification and Characterization of Recombinant Cystic Fibrosis Transmembrane Conductance Regulator from Chinese Hamster Ovary and Insect Cells

Modification of an adenoviral vector with biologically selected peptides : A novel strategy for gene delivery to cells of choice

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