Retinal gene therapy : methods and protocols /

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Bibliographic Details
Imprint:New York, NY : Humana Press : Springer, [2018]
©2018
Description:1 online resource (xvi, 380 pages) : illustrations (some color)
Language:English
Series:Methods in molecular biology ; v. 1715
Methods in molecular biology (Clifton, N.J.) ; v. 1715.
Subject:
Format: E-Resource Book
URL for this record:http://pi.lib.uchicago.edu/1001/cat/bib/11389354
Hidden Bibliographic Details
Other authors / contributors:Boon, Camiel J. F., editor.
Wijnholds, Jan, editor.
ISBN:9781493975228
1493975226
9781493975211
1493975218
Digital file characteristics:text file PDF
Notes:Includes bibliographical references and index.
Online resource; title from PDF title pate (SpringerProtocol, viewed December 5, 2017).
Other form:Print version: Retinal gene therapy . New York: Springer, 2018 9781493975211
Table of 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.