Retinal gene therapy methods and protocols edited by Livia S. Carvalho

Format:

Book

Contributor:

Carvalho, Livia S., editor.

Series:

Methods in molecular biology ; v. 3023.

Methods in molecular biology 1940-6029 3023

Language:

English

Subjects (All):

Eye--Diseases--Gene therapy.

Eye.

Retinal degeneration--Gene therapy.

Retinal degeneration.

Eye--Diseases.

Eye Diseases.

Retinal Degeneration.

Genetic Therapy--methods.

Medical Subjects:

Eye Diseases.

Retinal Degeneration.

Genetic Therapy--methods.

Genre:

Laboratory manuals.

Physical Description:

1 online resource

Place of Publication:

New York, NY Humana Press [2026]

Summary:

"This volume covers the latest advancements in the study of retinal gene therapy and addresses critical aspects of the gene therapy development pipeline. The chapters in this book are organized into four parts. Part One focuses on vector development and design, and talks about the latest approaches used to enhance the specificity and efficiency of gene delivery to retinal cells. Part Two presents a series of in vitro assays that are important for validating retinal gene therapies. Part Three discusses in vivo proof-of-concept studies and therapy validation in animal models. Part Four addresses clinical validation and presents methods for assessing visual function in human subjects undergoing retinal gene therapy. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Cutting-edge and comprehensive, Retinal Gene Therapy: Methods and Protocols is a valuable resource for researchers, clinicians, and students who are interested in learning more about this rapidly evolving field and looking for new research directions and collaborations across disciplines"-- Springer Nature Link

Contents:

Selecting novel retino-tropic AAV capsids by directed evolution / Adrian Westhaus and Leszek Lisowski

Evaluation of CRISPR/CasRx‑mediated VEGF mRNA knockdown in mouse retina / Satheesh Kumar, Da Zhao, Vickie H. Y. Wong, Bang V. Bui, and Guei‑Sheung Liu

Optimized retinal cell dissociation for downstream analysis of cone photoreceptors / Alicia A. Brunet, Paula I. Fuller‑Carter, and Livia S. Carvalho

Development of in vitro potency assays for gene therapy products targeting cGMP‑related retinopathies / Marina Pavlou and Stylianos Michalakis

Quantification of drusen‑like deposits in human pluripotent stem cell‑derived retinal pigment epithelium cells using confocal microscopy / Jenna Hall, Shane Cheung, Kalyan Shobhana, Ellie Cho, Grace E. Lidgerwood, Daniel Paull, and Alice Pébay

Ex vivo culture and formalin‑fixed paraffin embedding of murine retinal explants / Susan Haller, Rebecca M. Marton, and Eliah R. Shamir

Culture of human retinal explants as an ex vivo model for retinal gene therapy / Almaqdad Alsalloum, Madina Khubetsova, Olga Mityaeva, and Pavel Volchkov

Electrophysiological characterization of photoreceptor‑like cells in retinal organoids using whole‑cell patch clamp method / Almaqdad Alsalloum, Daria Kolotova, Alexey Malyshev, and Pavel Volchkov

Generation and characterization of patient‑derived human pluripotent stem cell lines for retinal gene therapy / Nathan Ehrenreich, Thibaud Metais, and Marcela Garita‑Hernandez

Transduction of AAV vectors in iPSC‑derived retinal organoids / Deborah Aubin and Anai Gonzalez‑Cordero

Intravitreal injections in mouse models of eye disease / Annie L. Miller, Yashvi Bhatt, and Livia S. Carvalho

Behavioral assessment of vision in dogs using a four‑alternative, choice‑based testing device / Freya M. Mowat and Simon M. Petersen‑Jones

Behavioral vision testing using an automated observational tracking system (VisioBox) to obtain a measure of visual acuity in larval zebrafish / Emily L. Allan and Jessica K. Mountford

Design and validation of mirtron‑mediated RNA interference for gene therapy / Qingyun Zheng, Harry O. Orlans, Federica Staurenghi, Michelle E. McClements, and Robert E. MacLaren

Applications of fundus‑tracked perimetry in the assessment of visual sensitivity / Alexis Ceecee Britten‑Jones, Janise Dagelet, and Lauren N. Ayton

Assessing visual function in retinal gene therapy / Andrew J. Anderson, Michael Bach, and Ruth E. Hogg

Notes:

Includes bibliographical references and index

Online resource; title from PDF title page (Springer Nature Link, viewed April 28, 2026)

Other Format:

Print version Retinal gene therapy

ISBN:

9781071651988

1071651986

OCLC:

1586994567

Access Restriction:

Restricted for use by site license