AAV-PHP.eB transduces both the inner and outer retina with high efficacy in mice

Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset

(2021) Nancy J. Newman et al.   OPHTHALMOLOGY

Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders

(2021) Marina Pavlou et al.   EMBO Molecular Medicine

Next-Generation Sequencing Applications for Inherited Retinal Diseases

(2021) Adrian Dockery et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR

(2020) Jasmina Cehajic-Kapetanovic et al.   NATURE MEDICINE

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

Findings from a Genotyping Study of over 1000 People with Inherited Retinal Disorders in Ireland

(2020) Laura Whelan et al.   Genes

Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy

(2020) Sanford L. Boye et al.   MOLECULAR THERAPY

Gene replacement therapy provides benefit in an adult mouse model of Leigh syndrome

(2020) Robin Reynaud-Dulaurier et al.   BRAIN

Non-photoreceptor Expression of Tulp1 May Contribute to Extensive Retinal Degeneration in Tulp1-/- Mice

(2020) Arpad Palfi et al.   Frontiers in Neuroscience

Cell Types of the Human Retina and Its Organoids at Single-Cell Resolution

(2020) Cameron S. Cowan et al.   CELL

Novel 199 base pair NEFH promoter drives expression in retinal ganglion cells

(2020) Sophia Millington-Ward et al.   Scientific Reports

CNS transduction benefits of AAV-PHP.eB over AAV9 are dependent on administration route and mouse strain.

(2020) Sophie N. Mathiesen et al.   Molecular Therapy-Methods & Clinical Development

AAVcis-regulatory sequences are correlated with ocular toxicity

(2019) Wenjun Xiong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier

(2019) Juliette Hordeaux et al.   MOLECULAR THERAPY

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

(2019) Albert M. Maguire et al.   OPHTHALMOLOGY

AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice

(2019) William A. Liguore et al.   MOLECULAR THERAPY

Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids

(2019) Qin Huang et al.   PLoS One

Ly6a Differential Expression in Blood–Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B

(2019) Ana Rita Batista et al.   HUMAN GENE THERAPY

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Safety of rAAV2/2- ND4 Gene Therapy for Leber Hereditary Optic Neuropathy

(2018) Catherine Vignal et al.   OPHTHALMOLOGY

OPA1: How much do we know to approach therapy?

(2018) Valentina Del Dotto et al.   PHARMACOLOGICAL RESEARCH

Inherited Retinal Degenerations: Current Landscape and Knowledge Gaps

(2018) Jacque L. Duncan et al.   Translational Vision Science & Technology

Dosage thresholds and influence of transgene cassette in AAV-related toxicity

(2018) Hanen Khabou et al.   HUMAN GENE THERAPY

AAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome

(2017) I Di Meo et al.   GENE THERAPY

Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups

(2017) Alisha M. Gruntman et al.   HUMAN GENE THERAPY

Toward an elucidation of the molecular genetics of inherited retinal degenerations

(2017) G. Jane Farrar et al.   HUMAN MOLECULAR GENETICS

Genetics of glaucoma

(2017) Janey L. Wiggs et al.   HUMAN MOLECULAR GENETICS

Application of polyploid adeno-associated virus vectors for transduction enhancement and neutralizing antibody evasion

(2017) Zheng Chai et al.   JOURNAL OF CONTROLLED RELEASE

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

A New Promoter Allows Optogenetic Vision Restoration with Enhanced Sensitivity in Macaque Retina

(2017) Antoine Chaffiol et al.   MOLECULAR THERAPY

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

A Novel Retinal Ganglion Cell Promoter for Utility in AAV Vectors

(2017) Killian S. Hanlon et al.   Frontiers in Neuroscience

Target 5000: Target Capture Sequencing for Inherited Retinal Degenerations

(2017) Adrian Dockery et al.   Genes

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

Gene Therapy for Leber Hereditary Optic Neuropathy

(2016) William J. Feuer et al.   OPHTHALMOLOGY

Identification of an Alternative Splicing Product of the Otx2 Gene Expressed in the Neural Retina and Retinal Pigmented Epithelial Cells

(2016) Christo Kole et al.   PLoS One

The Expression Pattern of Systemically Injected AAV9 in the Developing Mouse Retina Is Determined by Age

(2015) Leah C Byrne et al.   MOLECULAR THERAPY

Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho−/− mouse

(2015) Arpad Palfi et al.   Molecular Therapy-Methods & Clinical Development

Vector platforms for gene therapy of inherited retinopathies

(2014) Ivana Trapani et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Combined Rod and Cone Transduction by Adeno-Associated Virus 2/8

(2013) Anna Manfredi et al.   HUMAN GENE THERAPY

Tropisms of AAV for Subretinal Delivery to the Neonatal Mouse Retina and Its Application for In Vivo Rescue of Developmental Photoreceptor Disorders

(2013) Satoshi Watanabe et al.   PLoS One

Phototoxic Action Spectrum on a Retinal Pigment Epithelium Model of Age-Related Macular Degeneration Exposed to Sunlight Normalized Conditions

(2013) Emilie Arnault et al.   PLoS One

Intravitreal delivery of AAV-NDI1 provides functional benefit in a murine model of Leber hereditary optic neuropathy

(2012) Naomi Chadderton et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Fatal breathing dysfunction in a mouse model of Leigh syndrome

(2012) Albert Quintana et al.   JOURNAL OF CLINICAL INVESTIGATION

Transcriptional code and disease map for adult retinal cell types

(2012) Sandra Siegert et al.   NATURE NEUROSCIENCE

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

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

(2011) D Dalkara 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

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

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

Inhibition of Choroidal Neovascularization in a Nonhuman Primate Model by Intravitreal Administration of an AAV2 Vector Expressing a Novel Anti-VEGF Molecule

(2010) Michael Lukason et al.   MOLECULAR THERAPY

High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

(2009) E Ayuso et al.   GENE THERAPY

Adeno-Associated Virus-Mediated Rhodopsin Replacement Provides Therapeutic Benefit in Mice with a Targeted Disruption of the Rhodopsin Gene

(2009) Arpad Palfi et al.   HUMAN GENE THERAPY

Novel AAV serotypes for improved ocular gene transfer

(2008) Corinna Lebherz et al.   JOURNAL OF GENE MEDICINE

Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

(2008) Jeannette Bennicelli et al.   MOLECULAR THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY