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Adeno-Associated Viral Vectors as a Tool for Large Gene Delivery to the Retina

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An Overview of the Genetics of ABCA4 Retinopathies, an Evolving Story

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Assessment of Adeno-Associated Virus Serotype Tropism in Human Retinal Explants

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Could artificial intelligence revolutionize the development of nanovectors for gene therapy and mRNA vaccines?

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Vectors and Gene Delivery to the Retina

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Retinal transduction profiles by high-capacity viral vectors

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Dual <i>ABCA4</i> -AAV Vector Treatment Reduces Pathogenic Retinal A2E Accumulation in a Mouse Model of Autosomal Recessive Stargardt Disease

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Stargardt disease and progress in therapeutic strategies

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New frontiers to cure Alport syndrome: COL4A3 and COL4A5 gene editing in podocyte-lineage cells

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Has retinal gene therapy come of age? From bench to bedside and back to bench

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Promising and delivering gene therapies for vision loss

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Therapy Approaches for Stargardt Disease

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Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor

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Therapeutic homology-independent targeted integration in retina and liver

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Hair Cell Transduction Efficiency of Single- and Dual-AAV Serotypes in Adult Murine Cochleae

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

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Contemporary Animal Models For Human Gene Therapy Applications

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Ataluren for the Treatment of Usher Syndrome 2A Caused by Nonsense Mutations

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Dual-AAV delivery of large gene sequences to the inner ear

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Targeting mechanosensitive endothelial TXNDC5 to stabilize eNOS and reduce atherosclerosis in vivo

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A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome

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Targeting the photoreceptor cilium for the treatment of retinal diseases

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Gene therapy for inherited retinal degenerations: initial successes and future challenges

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Dual AAV Vectors for Stargardt Disease

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Outer Retinal Changes Including the Ellipsoid Zone Band in Usher Syndrome 1B due to MYO7A Mutations

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CRISPR/Cas9 editing of the MYO7A gene in rhesus macaque embryos to generate a primate model of Usher syndrome type 1B

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Clinical and Translational Landscape of Viral Gene Therapies

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Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B

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Split AAV8 Mediated ABCA4 Expression for Gene Therapy of Mouse Stargardt Disease (STGD1)

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Retina-directed gene therapy: Achievements and remaining challenges

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Ocular gene therapy for choroideremia: clinical trials and future perspectives

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Split AAV-Mediated Gene Therapy Restores Ureagenesis in a Murine Model of Carbamoyl Phosphate Synthetase 1 Deficiency

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Assessment of Therapeutic Potential of a Dual AAV Approach for Duchenne Muscular Dystrophy

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Assessment of AAV Dual Vector Safety in the <i>Abca4^−/−</i> Mouse Model of Stargardt Disease

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A versatile laser-induced porcine model of outer retinal and choroidal degeneration for preclinical testing

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Extending AAV Packaging Cargo through Dual Co-Transduction: Efficient Protein Trans-Splicing at Low Vector Doses

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Updates on Emerging Interventions for Autosomal Recessive ABCA4-Associated Stargardt Disease

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Retinal gene therapy: current progress and future prospects

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Advancing precision medicines for ocular disorders: Diagnostic genomics to tailored therapies

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Noninvasive Imaging and Correlative Histology of Cone Photoreceptor Structure in the Pig Retina

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Expression of two major isoforms of MYO7A in the retina: Considerations for gene therapy of Usher syndrome type 1B

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Clinical applications of retinal gene therapies

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Developing New Vectors for Retinal Gene TherapyPreprint

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Co-transduction of dual-adeno-associated virus vectors in the neonatal and adult mouse utricles

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Split otoferlins reunited

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Current approaches for Usher syndrome disease models and developing therapies

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Targeting Age-Related Neurodegenerative Diseases by AAV-Mediated Gene Therapy

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AAV Vector-Based Gene Therapy, Progress and Current Challenges

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Clinical Observation and Genotype-Phenotype Analysis of ABCA4- Related Hereditary Retinal Degeneration before Gene Therapy

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Gene therapy for inherited retinal diseases

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Key challenges in developing a gene therapy for Usher syndrome: machine-assisted scoping review

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Precision gene therapy: Tailoring rAAV-mediated gene therapies for inherited retinal dystrophies (IRDs)

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Translational readthrough using TRIDs - Achievements and challenges for the treatment of inherited retinal disorders

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Identifying and Overcoming Challenges in Developing Effective Treatments for Usher 1B: A Workshop Report

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Virus Vectors for Optogenetic Prosthetization of the Retina

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Improved protocol for histological and histopathological preparation of large eyes

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The potential use of anti-codon engineered tRNAs (ACE-tRNAs) to treat nonsense variants causing inherited retinal diseases

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Gene Therapy, Diet and Drug Approaches to Treating Inherited Retinal Disease

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AAV Capsid Screening for Translational Pig Research Using a Mouse Xenograft Liver ModelPreprint

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Evolution dirigée de virus adéno-associés pour un transfert de gène efficace dans le système visuel

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Adeno-associated virus in the liver : natural history of the infection and consequences in tumor development

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Better understanding of the ON-bipolar cell signaling cascade and development of therapies for congenital stationary night blindness

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Trends in the Engineering of Adeno-Associated Virus (AAV) for Precision Gene Delivery to the Central Nervous System (CNS)

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Effective delivery of large genes to the retina by dual AAV vectors

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Research Article Effective delivery of large genes to the retina by dual AAV vectors

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309. Optimization of Dual AAV Vectors for Gene Therapy of Inherited Retinal Diseases

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Dual AAV Vectors for Stargardt Disease

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Expanding AAV cargo capacity for gene therapy of Stargardt disease

Trapani, I. (2014)