Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9

Immune Responses to AAV in Clinical Trials

(2011) Federico Mingozzi et al.   CURRENT GENE THERAPY

Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system

(2011) Ahad A. Rahim et al.   FASEB JOURNAL

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

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

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

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

(2010) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

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

(2010) T. Leveillard et al.   Science Translational Medicine

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

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

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

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

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

microRNAs - powerful repression comes from small RNAs

(2009) Cong Ma et al.   Science in China. Series C, Life sciences / Chinese Academy of Sciences

Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising

(2008) J W Bainbridge et al.   GENE THERAPY

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

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

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

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

(2008) Albert M. Maguire 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