Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates

Imaging Light Responses of Foveal Ganglion Cells in the Living Macaque Eye

(2014) L. Yin et al.   JOURNAL OF NEUROSCIENCE

Focal damage to macaque photoreceptors produces persistent visual loss

(2013) Jennifer M. Strazzeri et al.   EXPERIMENTAL EYE RESEARCH

AAV-mediated gene delivery in Dp71-null mouse model with compromised barriers

(2013) Ophélie Vacca et al.   GLIA

Strong Cortical and Spinal Cord Transduction After AAV7 and AAV9 Delivery into the Cerebrospinal Fluid of Nonhuman Primates

(2013) Lluis Samaranch et al.   HUMAN GENE THERAPY

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

(2013) D. Dalkara et al.   Science Translational Medicine

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

(2013) F. Mingozzi et al.   Science Translational Medicine

Immunology of AAV-Mediated Gene Transfer in the Eye

(2013) Keirnan Willett et al.   Frontiers in Immunology

Intranasal Vaccination with AAV5 and 9 Vectors Against Human Papillomavirus Type 16 in Rhesus Macaques

(2012) Karen Nieto et al.   HUMAN GENE THERAPY

Effect of Alipogene Tiparvovec (AAV1-LPLS447X) on Postprandial Chylomicron Metabolism in Lipoprotein Lipase-Deficient Patients

(2012) André C. Carpentier et al.   JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

(2012) J. Bennett et al.   Science Translational Medicine

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID)

(2011) Mariell Jessup et al.   CIRCULATION

Immune Responses to AAV in Clinical Trials

(2011) Federico Mingozzi et al.   CURRENT GENE THERAPY

Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors

(2011) Lili Wang et al.   HUMAN GENE THERAPY

The Assembly-Activating Protein Promotes Capsid Assembly of Different Adeno-Associated Virus Serotypes

(2011) F. Sonntag et al.   JOURNAL OF VIROLOGY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency

(2010) Daniel Gaudet et al.   ATHEROSCLEROSIS SUPPLEMENTS

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

A viral assembly factor promotes AAV2 capsid formation in the nucleolus

(2010) F. Sonntag et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Safety and Efficacy of Subretinal Readministration of a Viral Vector in Large Animals to Treat Congenital Blindness

(2010) D. Amado et al.   Science Translational Medicine

Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse

(2009) T K Park et al.   GENE THERAPY

Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

(2009) Artur V. Cideciyan et al.   HUMAN GENE THERAPY

Changes in Adeno-Associated Virus-Mediated Gene Delivery in Retinal Degeneration

(2009) Kathleen D. Kolstad et al.   HUMAN GENE THERAPY

Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID Trial), a First-in-Human Phase 1/2 Clinical Trial

(2009) Brian E. Jaski et al.   JOURNAL OF CARDIAC FAILURE

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

Molecular Evolution of Adeno-associated Virus for Enhanced Glial Gene Delivery

(2009) James T Koerber et al.   MOLECULAR THERAPY

The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques

(2009) Lili Wang et al.   MOLECULAR THERAPY

A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Müller Cells

(2009) Ryan R. Klimczak et al.   PLoS One

Directed evolution of adeno-associated virus to an infectious respiratory virus

(2009) K. J. D. A. Excoffon et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Molecular Engineering of Viral Gene Delivery Vehicles

(2008) David V. Schaffer et al.   Annual Review of Biomedical Engineering

Intramuscular Administration of AAV1-Lipoprotein Lipase S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients

(2008) Erik S. Stroes et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Impact of Preexisting Vector Immunity on the Efficacy of Adeno-Associated Virus-Based HIV-1 Gag Vaccines

(2008) Jianping Lin et al.   HUMAN GENE THERAPY

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

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

DNA Shuffling of Adeno-associated Virus Yields Functionally Diverse Viral Progeny

(2008) James T Koerber et al.   MOLECULAR THERAPY

High-efficiency Transduction of the Mouse Retina by Tyrosine-mutant AAV Serotype Vectors

(2008) Hilda Petrs-Silva et al.   MOLECULAR THERAPY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

(2008) L. Zhong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA