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Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector

Molecular therapy, 2016-02, Vol.24 (2), p.287-297

2016

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Manufacturing of recombinant adeno-associated viral vectors for clinical trials

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16002-16002, Article 16002

2016

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Adeno-Associated Virus Vectorology, Manufacturing, and Clinical Applications

Methods in Enzymology, 2012, Vol.507, p.229-254

2012

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Production and characterization of adeno-associated viral vectors

Nature protocols, 2006-11, Vol.1 (3), p.1412-1428

2006

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Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Molecular therapy, 2012-02, Vol.20 (2), p.443-455

2012

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Packaging Capacity of Adeno-Associated Virus Serotypes: Impact of Larger Genomes on Infectivity and Postentry Steps

Journal of Virology, 2005-08, Vol.79 (15), p.9933-9944

2005

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Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial

Human gene therapy, 2015-02, Vol.26 (2), p.69-81

2015

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Single Amino Acid Changes Can Influence Titer, Heparin Binding, and Tissue Tropism in Different Adeno-Associated Virus Serotypes

Journal of Virology, 2006-11, Vol.80 (22), p.11393-11397

2006

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Separate Basic Region Motifs within the Adeno-Associated Virus Capsid Proteins Are Essential for Infectivity and Assembly

Journal of Virology, 2006-06, Vol.80 (11), p.5199-5210

2006

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Generation of Novel AAV Variants by Directed Evolution for Improved CFTR Delivery to Human Ciliated Airway Epithelium

Molecular therapy, 2009-12, Vol.17 (12), p.2067-2077

2009

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Gene therapy approaches for equine osteoarthritis

Frontiers in veterinary science, 2022-09, Vol.9, p.962898-962898

2022

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Corrigendum: Gene therapy approaches for equine osteoarthritis

Frontiers in veterinary science, 2023-01, Vol.9, p.1117776-1117776

2023

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Surface-Exposed Adeno-Associated Virus Vp1-NLS Capsid Fusion Protein Rescues Infectivity of Noninfectious Wild-Type Vp2/Vp3 and Vp3-Only Capsids but Not That of Fivefold Pore Mutant Virions

Journal of Virology, 2007-08, Vol.81 (15), p.7833-7843

2007

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Optimization of scAAVIL-1ra In Vitro and In Vivo to Deliver High Levels of Therapeutic Protein for Treatment of Osteoarthritis

Molecular therapy. Nucleic acids, 2013-02, Vol.2 (2), p.e70-e70, Article e70

2013

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Safety and biodistribution assessment of sc-rAAV2.5IL-1Ra administered via intra-articular injection in a mono-iodoacetate-induced osteoarthritis rat model

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.15052, Article 15052

2016

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Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors

Gene therapy, 2023-04, Vol.30 (3-4), p.398-404

2023

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Adeno-associated Virus as a Gene Therapy Vector: Vector Development, Production and Clinical Applications

Advances in biochemical engineering, biotechnology, 2005, Vol.99, p.119-145

2005

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The Growing Promise of Gene Therapy Approaches

Cell and Gene Therapy Insights, 2018-11, Vol.4 (10), p.1043-1049

2018

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Prediction of adeno-associated virus neutralizing antibody activity for clinical application

Gene therapy, 2015-12, Vol.22 (12), p.984-992

2015

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Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector

Manufacturing of recombinant adeno-associated viral vectors for clinical trials

Adeno-Associated Virus Vectorology, Manufacturing, and Clinical Applications

Production and characterization of adeno-associated viral vectors

Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Packaging Capacity of Adeno-Associated Virus Serotypes: Impact of Larger Genomes on Infectivity and Postentry Steps

Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial

Single Amino Acid Changes Can Influence Titer, Heparin Binding, and Tissue Tropism in Different Adeno-Associated Virus Serotypes

Separate Basic Region Motifs within the Adeno-Associated Virus Capsid Proteins Are Essential for Infectivity and Assembly

Generation of Novel AAV Variants by Directed Evolution for Improved CFTR Delivery to Human Ciliated Airway Epithelium

Gene therapy approaches for equine osteoarthritis

Corrigendum: Gene therapy approaches for equine osteoarthritis

Surface-Exposed Adeno-Associated Virus Vp1-NLS Capsid Fusion Protein Rescues Infectivity of Noninfectious Wild-Type Vp2/Vp3 and Vp3-Only Capsids but Not That of Fivefold Pore Mutant Virions

Optimization of scAAVIL-1ra In Vitro and In Vivo to Deliver High Levels of Therapeutic Protein for Treatment of Osteoarthritis

Safety and biodistribution assessment of sc-rAAV2.5IL-1Ra administered via intra-articular injection in a mono-iodoacetate-induced osteoarthritis rat model

Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors

Adeno-associated Virus as a Gene Therapy Vector: Vector Development, Production and Clinical Applications

The Growing Promise of Gene Therapy Approaches

Prediction of adeno-associated virus neutralizing antibody activity for clinical application

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