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

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
H\'e9ne 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
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 L\'e9eillard, G\'e9aldine Puel, and Najate Ait-Ali
Glucose metabolism
Constance Cepko and Yunlun Xue
Optogenetics 1
Botond Roska and Jos?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
Christelle Monville, Olivier Goureau, and Karim Ben M'Barek
Strategies for regeneration
Tom Reh

Preparing Clinical trials, and outcome measures
Rodent models
Serge Picaud & Val\'e9ie 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 Authi\'e9R
Current status of clinical trials design and outcomes
Jacque Duncan and Eyal Banin

Index