Table of contents
About this book
This detailed volume describes a spectrum of methods and protocols that can be used for the bench-to-bedside development and evaluation of retinal gene therapy. Methods for the successful delivery of these gene therapy vector systems to the retina are examined, as well as assays to test the efficacy in vitro in cell cultures, for gene augmentation and gene editing in vivo on rodents, pigs, and monkey retinas, and on human retinal explants as well as in human clinical studies. Written for the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible protocols, and tips on troubleshooting and avoiding known pitfalls.
Authoritative and practical, Retinal Gene Therapy: Methods and Protocols provides a wide range of readers from students to research experts with vital information on ocular gene therapy vector technology, in vitro and in vivo biological assays, and clinical protocols, to promote further studies for the benefit of children and adults with inherited retinal disease.
Gene therapy vector Retinal cell types Gene augmentation Cell cultures Gene silencing Vector production AAVs
Editors and affiliations
- DOI https://doi.org/10.1007/978-1-4939-7522-8
- Copyright Information Springer Science+Business Media LLC 2018
- Publisher Name Humana Press, New York, NY
- eBook Packages Springer Protocols
- Print ISBN 978-1-4939-7521-1
- Online ISBN 978-1-4939-7522-8
- Series Print ISSN 1064-3745
- Series Online ISSN 1940-6029
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