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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Bibliographic Details
Group Author: Boon, Camiel J. F; Wijnholds, Jan
Published: Humana Press : Springer,
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 /