This volume covers the latest advancements in the study of retinal gene therapy and addresses critical aspects of the gene therapy development pipeline. The chapters in this book are organized into four parts. Part One focuses on vector development and design, and talks about the latest approaches used to enhance the specificity and efficiency of gene delivery to retinal cells. Part Two presents a series of in vitro assays that are important for validating retinal gene therapies. Part Three discusses in vivo proof-of-concept studies and therapy validation in animal models. Part Four addresses clinical validation and presents methods for assessing visual function in human subjects undergoing retinal gene therapy. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Cutting-edge and comprehensive, Retinal Gene Therapy: Methods and Protocols is a valuable resource for researchers, clinicians, and students who are interested in learning more about this rapidly evolving field and looking for new research directions and collaborations across disciplines.