AAV-Mediated Gene Supplementation Therapy in Achromatopsia Type 2: Preclinical Data on Therapeutic Time Window and Long-Term Effects

Gene therapy for achromatopsia

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

Advances in Gene Therapy for Diseases of the Eye

(2016) Lolita Petit et al.   HUMAN GENE THERAPY

Men and mice: Relating their ages

(2016) Sulagna Dutta et al.   LIFE SCIENCES

Emerging therapies for inherited retinal degeneration

(2016) H. P. N. Scholl et al.   Science Translational Medicine

AAV Vectors for FRET-Based Analysis of Protein-Protein Interactions in Photoreceptor Outer Segments

(2016) Elvir Becirovic et al.   Frontiers in Neuroscience

The cone dysfunction syndromes: Table 1

(2015) Jonathan Aboshiha et al.   BRITISH JOURNAL OF OPHTHALMOLOGY

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

Achromatopsia: On the Doorstep of a Possible Therapy

(2015) Ditta Zobor et al.   OPHTHALMIC RESEARCH

Scale Adjustments to Facilitate Two-Dimensional Measurements in OCT Images

(2015) Marina Garcia Garrido et al.   PLoS One

Identification of a Common Non-Apoptotic Cell Death Mechanism in Hereditary Retinal Degeneration

(2014) Blanca Arango-Gonzalez et al.   PLoS One

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

(2013) Anna Manfredi et al.   HUMAN GENE THERAPY

Gene replacement therapy for retinal CNG channelopathies

(2013) Christian Schön et al.   MOLECULAR GENETICS AND GENOMICS

Transient Photoreceptor Deconstruction by CNTF Enhances rAAV-Mediated Cone Functional Rescue in Late Stage CNGB3-Achromatopsia

(2013) András M Komáromy et al.   MOLECULAR THERAPY

AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina

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

A Nonsense Mutation in PDE6H Causes Autosomal-Recessive Incomplete Achromatopsia

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

Gene therapy restores vision and delays degeneration in the CNGB1-/- mouse model of retinitis pigmentosa

(2012) S. Koch et al.   HUMAN MOLECULAR GENETICS

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

(2012) Ji-jing Pang et al.   PLoS One

Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy

(2011) L. S. Carvalho et al.   HUMAN MOLECULAR GENETICS

Molecular mechanism for 3:1 subunit stoichiometry of rod cyclic nucleotide-gated ion channels

(2011) Noah G. Shuart et al.   Nature Communications

Gene therapy rescues cone function in congenital achromatopsia

(2010) András M. Komáromy et al.   HUMAN MOLECULAR 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

Achromatopsie

(2010) C.M. Poloschek et al.   OPHTHALMOLOGE

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

Vision tests in the mouse: Functional phenotyping with electroretinography

(2009) Naoyuki Tanimoto   Frontiers in Bioscience-Landmark

Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints

(2009) L H Vandenberghe et al.   GENE THERAPY

Gene therapy for red–green colour blindness in adult primates

(2009) Katherine Mancuso et al.   NATURE

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

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

A homologous genetic basis of the murine cpfl1 mutant and human achromatopsia linked to mutations in the PDE6C gene

(2009) B. Chang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression

(2008) Li Zhong et al.   VIROLOGY