Retinal Gene Therapy: Methods and Protocols

Author:   Camiel J.F. Boon ,  Jan Wijnholds
Publisher:   Humana Press Inc.
Edition:   Softcover reprint of the original 1st ed. 2018
Volume:   1715
ISBN:  

9781493985098


Pages:   380
Publication Date:   05 September 2018
Format:   Paperback
Availability:   Manufactured on demand   Availability explained
We will order this item for you from a manufactured on demand supplier.

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Retinal Gene Therapy: Methods and Protocols


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Author:   Camiel J.F. Boon ,  Jan Wijnholds
Publisher:   Humana Press Inc.
Imprint:   Humana Press Inc.
Edition:   Softcover reprint of the original 1st ed. 2018
Volume:   1715
Dimensions:   Width: 17.80cm , Height: 2.10cm , Length: 25.40cm
Weight:   0.751kg
ISBN:  

9781493985098


ISBN 10:   1493985094
Pages:   380
Publication Date:   05 September 2018
Audience:   Professional and scholarly ,  Professional & Vocational
Format:   Paperback
Publisher's Status:   Active
Availability:   Manufactured on demand   Availability explained
We will order this item for you from a manufactured on demand supplier.

Table of 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.- Recordingand 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.

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