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Retinal Gene Therapy

Overview of attention for book
Cover of 'Retinal Gene Therapy'

Table of Contents

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    Book Overview
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    Chapter 1 Small Scale Production of Recombinant Adeno-Associated Viral Vectors for Gene Delivery to the Nervous System
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    Chapter 2 Small and Micro-Scale Recombinant Adeno-Associated Virus Production and Purification for Ocular Gene Therapy Applications
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    Chapter 3 Design and Development of AAV-based Gene Supplementation Therapies for Achromatopsia and Retinitis Pigmentosa
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    Chapter 4 Development of Multigenic Lentiviral Vectors for Cell-Specific Expression of Antiangiogenic miRNAs and Protein Factors
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    Chapter 5 Design and In Vitro Use of Antisense Oligonucleotides to Correct Pre-mRNA Splicing Defects in Inherited Retinal Dystrophies
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    Chapter 6 Three-Dimensional Co-Culture Bioassay for Screening of Retinal Gene Delivery Systems
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    Chapter 7 Retinal Gene Therapy for Choroideremia: In Vitro Testing for Gene Augmentation Using an Adeno-Associated Viral (AAV) Vector
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    Chapter 8 In Vivo Electroporation of Developing Mouse Retina
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    Chapter 9 Methods for In Vivo CRISPR/Cas Editing of the Adult Murine Retina
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    Chapter 10 AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa
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    Chapter 11 Dual AAV Vectors for Stargardt Disease
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    Chapter 12 Optogenetic Retinal Gene Therapy with the Light Gated GPCR Vertebrate Rhodopsin
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    Chapter 13 CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa: A Brief Methodology
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    Chapter 14 In-Depth Functional Analysis of Rodents by Full-Field Electroretinography
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    Chapter 15 Advanced Ocular Injection Techniques for Therapy Approaches
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    Chapter 16 Neutralizing Antibodies Against Adeno-Associated Virus (AAV): Measurement and Influence on Retinal Gene Delivery
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    Chapter 17 Screening for Neutralizing Antibodies Against Natural and Engineered AAV Capsids in Nonhuman Primate Retinas
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    Chapter 18 Subretinal and Intravitreal Retinal Injections in Monkeys
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    Chapter 19 Production of iPS-Derived Human Retinal Organoids for Use in Transgene Expression Assays
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    Chapter 20 AAV Serotype Testing on Cultured Human Donor Retinal Explants
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    Chapter 21 Human Retinal Explant Culture for Ex Vivo Validation of AAV Gene Therapy
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    Chapter 22 Visual Acuity Testing Before and After Intravitreal Injection of rAAV2-ND4 in Patients
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    Chapter 23 Recording and Analysis of the Human Clinical Electroretinogram
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    Chapter 24 Recording and Analysis of Goldmann Kinetic Visual Fields
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    Chapter 25 Measuring Central Retinal Sensitivity Using Microperimetry
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    Chapter 26 Inspection of the Human Retina by Optical Coherence Tomography
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    Chapter 27 Vector Shedding and Immunogenicity Sampling for Retinal Gene Therapy
Attention for Chapter 9: Methods for In Vivo CRISPR/Cas Editing of the Adult Murine Retina
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Chapter title
Methods for In Vivo CRISPR/Cas Editing of the Adult Murine Retina
Chapter number 9
Book title
Retinal Gene Therapy
Published in
Methods in molecular biology, January 2018
DOI 10.1007/978-1-4939-7522-8_9
Pubmed ID
Book ISBNs
978-1-4939-7521-1, 978-1-4939-7522-8
Authors

Sandy S. Hung, Fan Li, Jiang-Hui Wang, Anna E. King, Bang V. Bui, Guei-Sheung Liu, Alex W. Hewitt, Hung, Sandy S., Li, Fan, Wang, Jiang-Hui, King, Anna E., Bui, Bang V., Liu, Guei-Sheung, Hewitt, Alex W.

Abstract

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein (Cas) is used by some bacteria and most archaea to protect against viral phage intrusion and has recently been adapted to allow for efficient editing of the mammalian genome. Whilst CRISPR/Cas-based technology has been used to modify genes in mammalian cells in vitro, delivery of CRISPR/Cas system into mammalian tissue and/or organs is more difficult and often requires additional vectors. With the use of adeno-associated virus (AAV) gene delivery system, active CRISPR/Cas enzyme can be maintained for an extended period of time and enable efficient editing of genome in the retina in vivo. Herein we outline the method to edit the genome in mouse retina using a dual AAV vector -mediated CRISPR/Cas9 system.

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X Demographics

The data shown below were collected from the profile of 1 X user who shared this research output. Click here to find out more about how the information was compiled.
Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 33 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 33 100%

Demographic breakdown

Readers by professional status Count As %
Researcher 6 18%
Student > Ph. D. Student 5 15%
Student > Master 4 12%
Student > Bachelor 3 9%
Student > Postgraduate 2 6%
Other 3 9%
Unknown 10 30%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 8 24%
Agricultural and Biological Sciences 5 15%
Medicine and Dentistry 3 9%
Neuroscience 2 6%
Psychology 1 3%
Other 3 9%
Unknown 11 33%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 1. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 01 December 2017.
All research outputs
#20,453,782
of 23,009,818 outputs
Outputs from Methods in molecular biology
#9,941
of 13,157 outputs
Outputs of similar age
#378,135
of 442,310 outputs
Outputs of similar age from Methods in molecular biology
#1,193
of 1,498 outputs
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So far Altmetric has tracked 13,157 research outputs from this source. They receive a mean Attention Score of 3.4. This one is in the 1st percentile – i.e., 1% of its peers scored the same or lower than it.
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