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Engineering adeno-associated virus vectors for gene therapy

Nature reviews. Genetics, 2020-04, Vol.21 (4), p.255-272

2020

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Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective

Human gene therapy, 2015-05, Vol.26 (5), p.257-265

2015

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The AAV Vector Toolkit: Poised at the Clinical Crossroads

Molecular therapy, 2012-04, Vol.20 (4), p.699-708

2012

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Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

Gene therapy, 2013-04, Vol.20 (4), p.450-459

2013

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AAV-Mediated Gene Therapy for Research and Therapeutic Purposes

Annual review of virology, 2014-11, Vol.1 (1), p.427-451

2014

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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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Adeno-associated Virus Serotypes: Vector Toolkit for Human Gene Therapy

Molecular therapy, 2006-09, Vol.14 (3), p.316-327

2006

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Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates

Molecular therapy, 2011-06, Vol.19 (6), p.1058-1069

2011

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Parkinson's Disease Gene Therapy: Success by Design Meets Failure by Efficacy

Molecular therapy, 2014-03, Vol.22 (3), p.487-497

2014

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Viral vectors for gene delivery to the central nervous system

Neurobiology of disease, 2012-11, Vol.48 (2), p.179-188

2012

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Bound Protein- and Peptide-Based Strategies for Adeno-Associated Virus Vector-Mediated Gene Therapy: Where Do We Stand Now?

Human gene therapy, 2020-11, Vol.31 (21-22), p.1146-1154

2020

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Recombinant adeno-associated virus utilizes host cell nuclear import machinery to enter the nucleus

Journal of virology, 2014-04, Vol.88 (8), p.4132-4144

2014

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Cytoplasmic Trafficking, Endosomal Escape, and Perinuclear Accumulation of Adeno-Associated Virus Type 2 Particles Are Facilitated by Microtubule Network

Journal of Virology, 2012-10, Vol.86 (19), p.10462-10473

2012

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BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Blood, 2021-02, Vol.137 (6), p.763-774

2021

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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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Production and characterization of adeno-associated viral vectors

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

2006

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Sub-2 Å Ewald curvature corrected structure of an AAV2 capsid variant

Nature communications, 2018-09, Vol.9 (1), p.3628-11, Article 3628

2018

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Biosafety of recombinant adeno-associated virus vectors

Current gene therapy, 2013-12, Vol.13 (6), p.434-452

2013

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Enhancement of Adeno-Associated Virus Infection by Mobilizing Capsids into and Out of the Nucleolus

Journal of Virology, 2009-03, Vol.83 (6), p.2632-2644

2009

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Structure and Dynamics of Adeno-Associated Virus Serotype 1 VP1-Unique N-Terminal Domain and Its Role in Capsid Trafficking

Journal of Virology, 2013-05, Vol.87 (9), p.4974-4984

2013

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Mechanistic Insights into the Enhancement of Adeno-Associated Virus Transduction by Proteasome Inhibitors

Journal of Virology, 2013-12, Vol.87 (23), p.13035-13041

2013

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Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors

Human gene therapy, 2011-09, Vol.22 (9), p.1143-1153

2011

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Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles

Journal of Virology, 2012-08, Vol.86 (15), p.7752-7759

2012

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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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Structural Characterization of the Dual Glycan Binding Adeno-Associated Virus Serotype 6

Journal of Virology, 2010-12, Vol.84 (24), p.12945-12957

2010

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Engineering adeno-associated virus vectors for gene therapy

Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective

The AAV Vector Toolkit: Poised at the Clinical Crossroads

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

AAV-Mediated Gene Therapy for Research and Therapeutic Purposes

Adeno-Associated Virus Vectorology, Manufacturing, and Clinical Applications

Adeno-associated Virus Serotypes: Vector Toolkit for Human Gene Therapy

Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates

Parkinson's Disease Gene Therapy: Success by Design Meets Failure by Efficacy

Viral vectors for gene delivery to the central nervous system

Bound Protein- and Peptide-Based Strategies for Adeno-Associated Virus Vector-Mediated Gene Therapy: Where Do We Stand Now?

Recombinant adeno-associated virus utilizes host cell nuclear import machinery to enter the nucleus

Cytoplasmic Trafficking, Endosomal Escape, and Perinuclear Accumulation of Adeno-Associated Virus Type 2 Particles Are Facilitated by Microtubule Network

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

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

Production and characterization of adeno-associated viral vectors

Sub-2 Å Ewald curvature corrected structure of an AAV2 capsid variant

Biosafety of recombinant adeno-associated virus vectors

Enhancement of Adeno-Associated Virus Infection by Mobilizing Capsids into and Out of the Nucleolus

Structure and Dynamics of Adeno-Associated Virus Serotype 1 VP1-Unique N-Terminal Domain and Its Role in Capsid Trafficking

Mechanistic Insights into the Enhancement of Adeno-Associated Virus Transduction by Proteasome Inhibitors

Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors

Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles

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

Structural Characterization of the Dual Glycan Binding Adeno-Associated Virus Serotype 6

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