Retinal Gene Therapy

Small Scale Production of Recombinant Adeno-Associated Viral Vectors for Gene Delivery to the Nervous System.- Small and Micro-Scale Recombinant Adeno-Associated Virus Production and Purification for Ocular Gene Therapy Applications.- Design and Development of AAV-Based Gene Supplementation Therapies for Achromatopsia and Retinitis Pigmentosa.- Development of Multigenic Lentiviral Vectors for Cell-Specific Expression of Antiangiogenic miRNAs and Protein Factors.- Design and In Vitro Use of Antisense Oligonucleotides to Correct Pre-mRNA Splicing Defects in Inherited Retinal Dystrophies.- Three-Dimensional Co-Culture Bioassay for Screening of Retinal Gene Delivery Systems.- Retinal Gene Therapy for Choroideremia: In Vitro Testing for Gene Augmentation Using an Adeno-Associated Viral (AAV) Vector.- In Vivo Electroporation of Developing Mouse Retina.- Methods for In Vivo CRISPR/Cas Editing of the Adult Murine Retina.- AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa.- Dual AAV Vectors for Stargardt Disease.- Optogenetic Retinal Gene Therapy with the Light Gated GPCR Vertebrate Rhodopsin.- CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa: A Brief Methodology.- In-Depth Functional Analysis of Rodents by Full-Field Electroretinography.- Advanced Ocular Injection Techniques for Therapy Approaches.- Neutralizing Antibodies Against Adeno-Associated Virus (AAV): Measurement and Influence on Retinal Gene Delivery.- Screening for Neutralizing Antibodies against Natural and Engineered AAV Capsids in Non-Human Primate Retinas.- Subretinal and Intravitreal Retinal Injections in Monkeys.- Production of iPS-derived Human Retinal Organoids for Use in Transgene Expression Assays.- AAV Serotype Testing on Cultured Human Donor Retinal Explants.- Human Retinal Explant Culture for Ex-Vivo Validation of AAV Gene Therapy.- Visual Acuity Testing Before and After Intravitreal Injection of rAAV2- ND4 in Patients.- Recordingand Analysis of the Human Clinical Electroretinogram.- Recording and Analysis of Goldmann Kinetic Visual Fields.- Measuring Central Retinal Sensitivity Using Microperimetry.- Inspection of the Human Retina by Optical Coherence Tomography.- Vector Shedding and Immunogenicity Sampling for Retinal Gene Therapy.