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Retinal gene therapy : methods and protocols

Author: Camiel J F Boon; Jan Wijnholds
Publisher: New York, NY : Humana Press ; Springer, [2018] ©2018
Series: Methods in molecular biology (Clifton, N.J.), v. 1715.
Edition/Format:   eBook : Document : EnglishView all editions and formats
Summary:

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  Read more...

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Genre/Form: Electronic books
Laboratory Manuals
Additional Physical Format: Print version:
Retinal gene therapy .
New York: Springer, 2018
Material Type: Document, Internet resource
Document Type: Internet Resource, Computer File
All Authors / Contributors: Camiel J F Boon; Jan Wijnholds
ISBN: 9781493975228 1493975226 9781493975211 1493975218
OCLC Number: 1013933210
Description: 1 online resource (xvi, 380 pages) : illustrations (some color)
Contents: 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 --
Small and micro-scale recombinant adeno-associated virus production and purification for ocular gene therapy applications / Christopher A. Reid and Daniel M. Lipinski --
Design and development of AAV-based gene supplementation therapies for achromatopsia and retinitis pigmentosa / Christian Schön, Elvir Becirovic, Martin Biel, and Stylianos Michalakis --
Development of multigenic lentiviral vectors for cell-specific expression of antiangiogenic miRNAs and protein factors / Anne Louise Askou and Thomas J. Corydon --
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 --
Three-dimensional co-culture bioassay for screening of retinal gene delivery systems / Ding Wen Chen, Kathleen Pauloff, and Marianna Foldvari --
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 --
In vivo electroporation of developing mouse retina / Jimmy de Melo and Seth Blackshaw --
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 --
AAV gene augmentation therapy for CRB1-associated retinitis pigmentosa / C. Henrique Alves and Jan Wijnholds --
Dual AAV vectors for stargardt disease / Ivana Trapani--
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 --
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 --
In-depth functional analysis of rodents by full-field electroretinography / Vithiyanjali Sothilingam, Regine Mühlfriedel, Naoyuki Tanimoto, and Mathias W. Seeliger --
Advanced ocular injection techniques for therapy approaches / Regine Mühlfriedel, Marina Garcia Garrido, Christine Wallrapp, and Mathias W. Seeliger --
Neutralizing antibodies against adeno-associated virus (AAV) : measurement and influence on retinal gene delivery / Mélissa Desrosiers and Deniz Dalkara --
Screening for neutralizing antibodies against natural and engineered AAV capsids in nonhuman primate retinas / Timothy P. Day, Leah C. Byrne, John G. Flannery, and David V. Schaffer --
Subretinal and intravitreal retinal injections in monkeys / Daniyar Dauletbekov, K. Ulrich Bartz-Schmidt, and M. Dominik Fischer --
Production of iPS-derived human retinal organoids for use in transgene expression assays / Peter M. Quinn, Thilo M. Buck, Charlotte Ohonin, Harald M.M. Mikkers, and Jan Wijnholds --
AAV serotype testing on cultured human donor retinal explants / Thilo M. Buck, Lucie P. Pellissier, Rogier M. Vos, Elon H.C. van Dijk, Camiel J.F. Boon, and Jan Wijnholds --
Human retinal explant culture for ex vivo validation of AAV gene therapy / Harry O. Orlans, Thomas L. Edwards, Samantha R. De Silva, Maria I. Patrício, and Robert E. MacLaren --
Visual acuity testing before and after intravitreal injection of rAAV2-ND4 in patients / Bin Li and Chenmian Wu --
Recording and analysis of the human clinical electroretinogram / Mathieu Gauvin, Allison L. Dorfman, and Pierre Lachapelle --
Recording and analysis of goldmann kinetic visual fields / Mays Talib, Gislin Dagnelie, and Camiel J.F. Boon --
Measuring central retinal sensitivity using microperimetry / Mays Talib, Jasleen K. Jolly, and Camiel J.F. Boon --
Inspection of the human retina by optical coherence tomography / Thomas Theelen and Michel M. Teussink --
Vector shedding and immunogenicity sampling for retinal gene therapy / Alun R. Barnard, Anna N. Rudenko, and Robert E. MacLaren.
Series Title: Methods in molecular biology (Clifton, N.J.), v. 1715.
Responsibility: edited by Camiel J.F. Boon, Jan Wijnholds.

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