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Retinal Disorders: Genetic Approaches to Diagnosis and Treatment, Second Edition


Subject Area(s):  Gene TherapyGeneticsNeurobiology

Edited by Eyan Banin, Hadassah-Hebrew University Medical Center, Jerusalem; Jean Bennett, University of Pennsylvania Perelman School of Medicine; Jacque L. Duncan, University of California, San Francisco; Botond Roska, IOB, Basel, Switzerland; José-Alain Sahel, University of Pittsburgh School of Medicine

Due May 2023 • 400 pages (approx.), illustrated, index
Hardcover • $135 94.50
ISBN  978-1-621824-62-6
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  •     Description    
  •     Contents    

Description

The retina is a layer of neural tissue that lines the inner eye and captures visual stimuli. This volume provides an update on retinal diseases, describing the genetic and pathophysiological basis of the diseases and advances in strategies to treat them.

Contents

Preliminary
Introduction
Genetics and pathophysiology of inherited retinal degenerations
An historical perspective on gene discovery in retinal dystrophies
Stephen P. Daiger
Gene poll studies
Kari Branham
World-wide carrier rates for IRD genes
Dror Sharon and Mor Hanany
Syndromic ciliopathy-related retinal dystrophies models
Héène Dollfus
High throughput organoid models
Magdalena Renner
Gene therapies
Overview (current status and future challenges)
Jean Bennett
Lessons learned from the RPE65 story
Albert Maguire and Jean Bennett
Bringing an innovative therapy to market
Kathy High + 1
Choroideremia
Robert MacLaren and Imran Yusuf
RPGR
Michel Michaelides, Michalis Georgiou, Saima Hasahem, and Robin Ali
Achromatopsia
Eyal Banin
From Bench to Bedside—Delivering Gene Therapy for Leber Hereditary Optic Neuropathy
Benson S. Chen and Patrick Yu-Wai-Man
Retinoschisis
Paul Sieving
Gene Therapy for Rhodopsin Mutations
Alfred S. Lewin and W. Clay Smith
Gene-independent approaches: Neuroprotection
Thierry Léveillard, Géraldine Puel, and Najate Ait-Ali
Glucose metabolism
Constance Cepko and Yunlun Xue
Optogenetics 1
Botond Roska and José-Alain Sahel
Optogenetics 2
John Flannery and Antonia Stefanov
Developing new vectors
Deniz Dalkara and Leah Byrne
Immune responses
Kathryn Pepple and Debarshi Mustafi
Therapeutic Gene Editing in Inherited Retinal Disorders
Jinjie Ling, Laura A. Jenny, Ashley Zhou, and Stephen H. Tsang
New medical therapies (related to use of retinal organoids)
Don Zack
Visual Prosthesis
Electronic Retinal Prostheses
Daniel Palanker
Cell-based therapies
Overview
Pete Coffey
iPSc Cell therapy 1
Masayo Takahashi
iPSc Cell therapy 2
Khapil Bharti and Fnu Ruchi
Retinal dystrophies (will focus on photoreceptor cell therapy)
Christelle Monville, Olivier Goureau, and Karim Ben M’Barek
Strategies for regeneration
Tom Reh
Preparing Clinical trials, and outcome measures
Rodent models
Serge Picaud & Valérie Fradot
Pathomechanisms and perspectives for neuroprotection
Francois Paquet-Durand
Large animal models: Dogs, cats, sheep, and cattle achromatopsia models
Simon Petersen-Jones and Andras Komaromy
Pig models
Maureen McCall
High resolution imaging
Joe Carroll and Niamh Wynne
The importance of natural history studies
Allison Ayala
Patient-reported outcomes
Todd Durham
Performance-based tests
Daniel Chung
Performance-based tests
Colas Authié
Current status of clinical trials design and outcomes
Jacque Duncan and Eyal Banin
Index