Retinal Gene Therapy : Methods and Protocols / edited by Camiel J.F. Boon, Jan Wijnholds.

Format:

Book

Contributor:

Boon, Camiel J. F., editor.

Wijnholds, Jan, editor.

SpringerLink (Online service)

Series:

Methods in molecular biology 1064-3745 ; 1715.

Methods in Molecular Biology, 1064-3745 ; 1715

Language:

English

Subjects (All):

Medicine.

Human genetics.

Biomedicine.

Human Genetics.

Local Subjects:

Biomedicine.

Human Genetics.

Physical Description:

1 online resource (XVI, 380 pages) : 54 illustrations, 44 illustrations in color.

Contained In:

Springer eBooks

Place of Publication:

New York, NY : Springer New York : Imprint: Humana Press, 2018.

System Details:

text file PDF

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 respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible protocols, and tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Retinal Gene Therapy: Methods and Protocols provides a wide range of readers from students to research experts with vital information on ocular gene therapy vector technology, in vitro and in vivo biological assays, and clinical protocols, to promote further studies for the benefit of children and adults with inherited retinal disease.

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

Development of Multigenic Lentiviral Vectors for Cell-Specific Expression of Antiangiogenic miRNAs and Protein Factors

Design and In Vitro Use of Antisense Oligonucleotides to Correct Pre-mRNA Splicing Defects in Inherited Retinal Dystrophies

Three-Dimensional Co-Culture Bioassay for Screening of Retinal Gene Delivery Systems

Retinal Gene Therapy for Choroideremia: In Vitro Testing for Gene Augmentation Using an Adeno-Associated Viral (AAV) Vector

In Vivo Electroporation of Developing Mouse Retina

Methods for In Vivo CRISPR/Cas Editing of the Adult Murine Retina

AAV Gene Augmentation Therapy for CRB1-Associated Retinitis Pigmentosa

Dual AAV Vectors for Stargardt Disease

Optogenetic Retinal Gene Therapy with the Light Gated GPCR Vertebrate Rhodopsin

CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa: A Brief Methodology

In-Depth Functional Analysis of Rodents by Full-Field Electroretinography

Advanced Ocular Injection Techniques for Therapy Approaches

Neutralizing Antibodies Against Adeno-Associated Virus (AAV): Measurement and Influence on Retinal Gene Delivery

Screening for Neutralizing Antibodies against Natural and Engineered AAV Capsids in Non-Human Primate Retinas

Subretinal and Intravitreal Retinal Injections in Monkeys

Production of iPS-derived Human Retinal Organoids for Use in Transgene Expression Assays

AAV Serotype Testing on Cultured Human Donor Retinal Explants

Human Retinal Explant Culture for Ex-Vivo Validation of AAV Gene Therapy

Visual Acuity Testing Before and After Intravitreal Injection of rAAV2- ND4 in Patients

Recording and Analysis of the Human Clinical Electroretinogram

Recording and Analysis of Goldmann Kinetic Visual Fields

Measuring Central Retinal Sensitivity Using Microperimetry

Inspection of the Human Retina by Optical Coherence Tomography

Vector Shedding and Immunogenicity Sampling for Retinal Gene Therapy.

Other Format:

Printed edition:

ISBN:

9781493975228

Access Restriction:

Restricted for use by site license.