Retinal Gene Therapy : Methods and Protocols - Boon, Camiel J.F.; Wijnholds, Jan.; SpringerLink (Online service) (New York, NY : Springer New York : Imprint: Humana, 2018)
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 -- Recording and 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.
Keywords: Human genetics; Human Genetics