Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia

Gene therapy for achromatopsia

(2017) Stylianos Michalakis et al.   JOURNAL OF GENE MEDICINE

Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep

(2017) Elisha Gootwine et al.   Human Gene Therapy Clinical Development

Novel mutations in the gene for α-subunit of retinal cone cyclic nucleotide-gated channels in a Japanese patient with congenital achromatopsia

(2016) Kazuki Kuniyoshi et al.   JAPANESE JOURNAL OF OPHTHALMOLOGY

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

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

Effects of Subretinal Gene Transfer at Different Time Points in a Mouse Model of Retinal Degeneration

(2016) Xufeng Dai et al.   PLoS One

Achromatopsia

(2015) Meredith H. Remmer et al.   CURRENT OPINION IN OPHTHALMOLOGY

Mutation of ATF6 causes autosomal recessive achromatopsia

(2015) Muhammad Ansar et al.   HUMAN GENETICS

Targeted Ablation of thePde6hGene in Mice Reveals Cross-species Differences in Cone and Rod Phototransduction Protein Isoform Inventory

(2015) Christina Brennenstuhl et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia

(2015) Eyal Banin et al.   MOLECULAR THERAPY

Mutations in the unfolded protein response regulator ATF6 cause the cone dysfunction disorder achromatopsia

(2015) Susanne Kohl et al.   NATURE GENETICS

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

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

Congenital Achromatopsia and Macular Atrophy Caused by a Novel RecessivePDE6CMutation (p.E591K)

(2015) Satoshi Katagiri et al.   OPHTHALMIC GENETICS

Investor Outlook: Significance of the Positive LCA2 Gene Therapy Phase III Results

(2015) Joshua Schimmer et al.   Human Gene Therapy Clinical Development

The Frequency-Response Electroretinogram Distinguishes Cone and Abnormal Rod Function in rd12 Mice

(2015) Xufeng Dai et al.   PLoS One

Trans-Corneal Subretinal Injection in Mice and Its Effect on the Function and Morphology of the Retina

(2015) Yan Qi et al.   PLoS One

Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies inin vitroandin vivomodels

(2014) Aaron Black et al.   JOURNAL OF GENE MEDICINE

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

(2014) Robert E MacLaren et al.   LANCET

Optimization of the Capsid of Recombinant Adeno-Associated Virus 2 (AAV2) Vectors: The Final Threshold?

(2013) George V. Aslanidi et al.   PLoS One

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

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

A Nonsense Mutation in PDE6H Causes Autosomal-Recessive Incomplete Achromatopsia

(2012) Susanne Kohl et al.   AMERICAN JOURNAL OF HUMAN GENETICS

AAV-Mediated Gene Therapy in Mouse Models of Recessive Retinal Degeneration

(2012) J.-J. Pang et al.   CURRENT MOLECULAR MEDICINE

AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia

(2012) Ji-jing Pang et al.   PLoS One

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey

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

Clinical and genetic investigation of a large Tunisian family with complete achromatopsia: identification of a new nonsense mutation in GNAT2 gene

(2010) Farah Ouechtati et al.   JOURNAL OF HUMAN GENETICS

Restoration of Cone Vision in the CNGA3−/− Mouse Model of Congenital Complete Lack of Cone Photoreceptor Function

(2010) Stylianos Michalakis et al.   MOLECULAR THERAPY

Long-term Retinal Function and Structure Rescue Using Capsid Mutant AAV8 Vector in the rd10 Mouse, a Model of Recessive Retinitis Pigmentosa

(2010) Ji-jing Pang et al.   MOLECULAR THERAPY

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

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

Homozygosity Mapping Reveals PDE6C Mutations in Patients with Early-Onset Cone Photoreceptor Disorders

(2009) Alberta A.H.J. Thiadens et al.   AMERICAN JOURNAL OF HUMAN GENETICS

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

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

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

(2009) Francesca Simonelli 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

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

Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: Effects of serotype and site of administration

(2007) Ji-jing Pang et al.   VISION RESEARCH