Retinal Gene Therapy: Methods and Protocols: 1715 (Methods in Molecular Biology) - Tapa blanda

Sinopsis

Part I: Retinal Gene Therapy Vector Production and Biological Assays In Vitro

1. Small Scale Production of Recombinant Adeno-Associated Viral Vectors for Gene Delivery to the Nervous System

Joost Verhaagen, Barbara Hobo, Erich M.E. Ehlert, Ruben Eggers, Joanna A. Korecka, Stefan A. Hoyng, Callan L. Attwell, Alan R. Harvey, and Matthew R.J. Mason

2. Small and Micro-Scale Recombinant Adeno-Associated Virus Production and Purification for Ocular Gene Therapy Applications

Christopher A. Reid and Daniel M. Lipinski

Christian Schön, Elvir Becirovic, Martin Biel, and Stylianos Michalakis

4. Development of Multigenic Lentiviral Vectors for Cell-Specific Expression of Antiangiogenic miRNAs and Protein Factors

Anne Louise Askou and Thomas J. Corydon

5. Design and In Vitro Use of Antisense Oligonucleotides to Correct Pre-mRNA Splicing Defects in Inherited Retinal Dystrophies

Alejandro Garanto and Rob W.J. Collin

6. Three-Dimensional Co-Culture Bioassay for Screening of Retinal Gene Delivery Systems

Ding Wen Chen, Kathleen Pauloff, and Marianna Foldvari

7. Retinal Gene Therapy for Choroideremia: In Vitro Testing for Gene Augmentation Using an Adeno-Associated Viral (AAV) Vector

Maria I. Patrício and Robert E. MacLaren

Part II: Assays for Gene Augmentation and Editing In Vivo on Rodent and Macaque Retina

8. In Vivo Electroporation of Developing Mouse Retina

9. Methods for In Vivo CRISPR/Cas Editing of the Adult Murine Retina

Sandy S. Hung, Fan Li, Jiang-Hui Wang, Anna E. King, Bang V. Bui, Guei-Sheung Liu, and Alex W. Hewitt

10. AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa

C. Henrique Alves and Jan Wijnholds

11. Dual AAV Vectors for Stargardt Disease

Ivana Trapani

12. Optogenetic Retinal Gene Therapy with the Light Gated GPCR Vertebrate Rhodopsin

Benjamin M. Gaub, Michael H. Berry, Meike Visel, Amy Holt, Ehud Y. Isacoff, and John G. Flannery

13. CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa: A Brief Methodology

Wen-Hsuan Wu, Yi-Ting Tsai, Sally Justus, Galaxy Y Cho, Jesse D Sengillo, Yu Xu, Thiago Cabral, Chyuan-Sheng Lin, Alexander G. Bassuk, Vinit B. Mahajan, and Stephen H. Tsang

14. In-Depth Functional Analysis of Rodents by Full-Field Electroretinography

Vithiyanjali Sothilingam, Regine Mühlfriedel, Naoyuki Tanimoto, and Mathias W. Seeliger

15. Advanced Ocular Injection Techniques for Therapy Approaches

Regine Mühlfriedel, Marina Garcia Garrido, Christine Wallrapp, and Mathias W. Seeliger

16. ­­Neutralizing Antibodies Against Adeno-Associated Virus (AAV): Measurement and Influence on Retinal Gene Delivery

Mélissa Desrosiers and Deniz Dalkara

17. Screening for Neutralizing Antibodies against Natural and Engineered AAV Capsids in Non-Human Primate Retinas

Timothy P. Day, Leah C. Byrne, John G. Flannery, and David V. Schaffer

18. Subretinal and Intravitreal Retinal Injections in Monkeys

Daniyar Dauletbekov, K. Ulrich Bartz-Schmidt,

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