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

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Functional and Anatomic Consequences of Subretinal Dosing in the Cynomolgus Macaque

(2011) T. Michael Nork   ARCHIVES OF OPHTHALMOLOGY

AAV-mediated photoreceptor transduction of the pig cone-enriched retina

(2011) C Mussolino et al.   GENE THERAPY

LiGluR Restores Visual Responses in Rodent Models of Inherited Blindness

(2011) Natalia Caporale et al.   MOLECULAR THERAPY

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

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

Preclinical Safety Evaluation of AAV2-sFLT01— A Gene Therapy for Age-related Macular Degeneration

(2010) Timothy K MacLachlan et al.   MOLECULAR THERAPY

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

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

Photoreceptor degeneration: genetic and mechanistic dissection of a complex trait

(2010) Alan F. Wright et al.   NATURE REVIEWS GENETICS

Genetic Reactivation of Cone Photoreceptors Restores Visual Responses in Retinitis Pigmentosa

(2010) V. Busskamp et al.   SCIENCE

Rod-Derived Cone Viability Factor for Treating Blinding Diseases: From Clinic to Redox Signaling

(2010) T. Leveillard 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

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

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

(2009) Deniz Dalkara et al.   MOLECULAR THERAPY

Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)

(2009) Steven J Gray 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

Noninvasive, In Vivo Assessment of Mouse Retinal Structure Using Optical Coherence Tomography

(2009) M. Dominik Fischer et al.   PLoS One

A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection

(2009) L. Yang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Remodeling of cone photoreceptor cells after rod degeneration in rd mice

(2008) Bin Lin et al.   EXPERIMENTAL EYE RESEARCH

Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injection

(2008) M Hellström et al.   GENE THERAPY

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

(2008) James T Koerber et al.   MOLECULAR THERAPY

Effect of Late-stage Therapy on Disease Progression in AAV-mediated Rescue of Photoreceptor Cells in the Retinoschisin-deficient Mouse

(2008) Andreas Janssen 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

Light-activated channels targeted to ON bipolar cells restore visual function in retinal degeneration

(2008) Pamela S Lagali et al.   NATURE NEUROSCIENCE

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

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