Retinal gene therapy : methods and protocols /
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...
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Group Author: | ; |
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Published: |
Humana Press : Springer,
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Publisher Address: | New York, NY : |
Publication Dates: | [2018] |
Literature type: | Book |
Language: | English |
Series: |
Methods in molecular biology,
volume 1715 Springer protocols, |
Subjects: | |
Summary: |
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 respec |
Carrier Form: | xvi, 380 pages : illustrations (some color) ; 26 cm. |
Bibliography: | Includes bibliographical references and index. |
ISBN: |
9781493975211 1493975218 |
Index Number: | QH506 |
CLC: | R774.105 |
Call Number: | R774.105/R438 |
Contents: |
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 / |