Insight into the mechanisms of enhanced retinal transduction by the engineered AAV2 capsid variant -7m8

Erratum: Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs

(2016) R F Boyd et al.   GENE THERAPY

Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors

(2016) Sanford L. Boye et al.   JOURNAL OF VIROLOGY

Targeting Channelrhodopsin-2 to ON-bipolar Cells With Vitreally Administered AAV Restores ON and OFF Visual Responses in Blind Mice

(2015) Emilie Macé et al.   MOLECULAR THERAPY

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

(2015) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Improvement and Decline in Vision with Gene Therapy in Childhood Blindness

(2015) Samuel G. Jacobson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter

(2014) T. Cronin et al.   EMBO Molecular Medicine

Retinoschisin gene therapy in photoreceptors, Müller glia or all retinal cells in the Rs1h−/− mouse

(2014) L C Byrne et al.   GENE THERAPY

Functional Analysis of the Putative Integrin Recognition Motif on Adeno-associated Virus 9

(2014) Shen Shen et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Viral-mediated RdCVF and RdCVFL expression protects cone and rod photoreceptors in retinal degeneration

(2014) Leah C. Byrne et al.   JOURNAL OF CLINICAL INVESTIGATION

Identification and Mutagenesis of the Adeno-Associated Virus 5 Sialic Acid Binding Region

(2014) Sandra Afione et al.   JOURNAL OF VIROLOGY

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

(2014) Robert E MacLaren et al.   LANCET

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach

(2013) F M Mowat et al.   GENE THERAPY

AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina

(2013) Luk H. Vandenberghe et al.   PLoS One

Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors

(2013) Christine N. Kay et al.   PLoS One

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

Heparin-binding correlates with increased efficiency of AAV1- and AAV6-mediated transduction of striated muscle, but negatively impacts CNS transduction

(2012) A L H Arnett et al.   GENE THERAPY

The Threefold Protrusions of Adeno-Associated Virus Type 8 Are Involved in Cell Surface Targeting as Well as Postattachment Processing

(2012) C. Raupp et al.   JOURNAL OF VIROLOGY

Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9

(2012) M. A. DiMattia et al.   JOURNAL OF VIROLOGY

Identification of the Galactose Binding Domain of the Adeno-Associated Virus Serotype 9 Capsid

(2012) C. L. Bell et al.   JOURNAL OF VIROLOGY

Universal Real-Time PCR for the Detection and Quantification of Adeno-Associated Virus Serotype 2-Derived Inverted Terminal Repeat Sequences

(2012) Christine Aurnhammer et al.   Human Gene Therapy Methods

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Novel adeno-associated viral vectors for retinal gene therapy

(2011) L H Vandenberghe et al.   GENE THERAPY

Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors

(2011) K Varadi et al.   GENE THERAPY

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

(2011) Hongwei Zhang et al.   MOLECULAR THERAPY

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

Peptide Ligands Incorporated into the Threefold Spike Capsid Domain to Re-Direct Gene Transduction of AAV8 and AAV9 In Vivo

(2011) Stefan Michelfelder et al.   PLoS One

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

(2011) L. H. Vandenberghe et al.   Science Translational Medicine

Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library

(2010) Y Ying et al.   GENE THERAPY

Novel Properties of Tyrosine-mutant AAV2 Vectors in the Mouse Retina

(2010) Hilda Petrs-Silva et al.   MOLECULAR THERAPY

Inner Limiting Membrane Barriers to AAV-mediated Retinal Transduction From the Vitreous

(2009) Deniz Dalkara et al.   MOLECULAR THERAPY

Molecular Characterization of the Heparin-Dependent Transduction Domain on the Capsid of a Novel Adeno-Associated Virus Isolate, AAV(VR-942)

(2008) M. Schmidt et al.   JOURNAL OF VIROLOGY

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

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

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

(2008) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA