Regenerative Medicine and Stem Cell Therapy for the Eye PDF
Preview Regenerative Medicine and Stem Cell Therapy for the Eye
Fundamental Biomedical Technologies Brian G. Ballios · Michael J. Young Editors Regenerative Medicine and Stem Cell Therapy for the Eye Fundamental Biomedical Technologies Series Editor Mauro Ferrari, Ph.D. The University of Texas Houston, TX, USA More information about this series at http://www.springer.com/series/7045 Brian G. Ballios • Michael J. Young Editors Regenerative Medicine and Stem Cell Therapy for the Eye Editors Brian G.Ballios Michael J. Young Department Ophthalmology Schepens Eye Research Institute and Vision Sciences Harvard Medical School University of Toronto Boston, MA, USA Toronto, ON, Canada ISSN 1559-7083 Fundamental Biomedical Technologies ISBN 978-3-319-98079-9 ISBN 978-3-319-98080-5 (eBook) https://doi.org/10.1007/978-3-319-98080-5 Library of Congress Control Number: 2018959866 © Springer Nature Switzerland AG 2018 This work is subject to copyright. All rights are reserved by the Publisher, whether the whole or part of the material is concerned, specifically the rights of translation, reprinting, reuse of illustrations, recitation, broadcasting, reproduction on microfilms or in any other physical way, and transmission or information storage and retrieval, electronic adaptation, computer software, or by similar or dissimilar methodology now known or hereafter developed. The use of general descriptive names, registered names, trademarks, service marks, etc. in this publication does not imply, even in the absence of a specific statement, that such names are exempt from the relevant protective laws and regulations and therefore free for general use. The publisher, the authors, and the editors are safe to assume that the advice and information in this book are believed to be true and accurate at the date of publication. Neither the publisher nor the authors or the editors give a warranty, express or implied, with respect to the material contained herein or for any errors or omissions that may have been made. The publisher remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. This Springer imprint is published by the registered company Springer Nature Switzerland AG The registered company address is: Gewerbestrasse 11, 6330 Cham, Switzerland Preface Recent advances in the field of stem cell biology have led to a new field that has become known as regenerative ophthalmology. The aim of this field is to preserve, repair, or replace ocular cells that have been injured or lost. We now better understand the complexity of how the eye develops, the cellular and regional heterogeneity that builds complex ocular tissues, and how these processes are disordered in disease. Ocular diseases affect the entire age spectrum, and loss of vision significantly affects the quality of life for those with blinding eye conditions. With our aging population, the burden of ocular disease is expected to rise over the next decades. As we enter the age of regenerative medicine, the eye has become the proving ground for first-in-man stem cell clinical trials. This is in part because of its accessibility for minimally invasive surgical approaches to deliver cellular therapeutics, the availability of multimodal behavioural, physiological, and imaging techniques, and the sheer number of patients that new treatments could potentially impact. The purpose of the book is to provide those interested in the field of ocular regenerative medicine with a perspective on the various ways in which stem cells are being applied in the development of potential cures for blinding eye diseases. The aim is to provide a review-level understanding of the types, sources, and applications of stem cells in regenerating tissues in various parts of the eye, with a perspective on the promise of stem cell applications, as well as challenges confronting the field. It is intended for those with a background in the biological and/or medical sciences. The field will depend increasingly on multidisciplinary collaboration between scientists, physicians, and engineers as we develop the tools needed to realize the full potential of stem cells to heal the eye. The scope of the text is broad and includes both preclinical and clinical applications, including stem cell-derived therapies based on endogenous tissue repair, stem cell transplantation/cell replacement therapy, the interface with gene therapy, and in vitro disease modelling. Applications in both anterior and posterior ocular disease are described, with a particular focus on diseases of the ocular surface/cornea/limbus, where stem cell transplantation has already found clinical application in patients, as well as diseases of the retina, where early clinical trials have begun for both RPE v vi Preface and photoreceptor replacement. Targets of these therapies include inherited retinal dystrophies as well as acquired diseases, such as age-related macular degeneration. As editors, we have had the pleasure of working with these researchers to bring this volume together. We extend our gratitude and thanks to all of the authors for their time and commitment to this volume and for providing their perspectives on this rapidly expanding field. Toronto, ON, Canada Brian G. Ballios Boston, MA, USA Michael J. Young Contents 1 Photoreceptor Cell Replacement Therapy from Stem Cells . . . . . . . . . 1 Gilbert Bernier 2 Human Pluripotent Stem Cells as In Vitro Models for Retinal Development and Disease . . . . . . . . . . . . . . . . . . . . . . . . . . . 17 Akshayalakshmi Sridhar, Kirstin B. Langer, Clarisse M. Fligor, Matthew Steinhart, Casey A. Miller, Kimberly T. Ho-A-Lim, Sarah K. Ohlemacher, and Jason S. Meyer 3 Bioengineered and Regenerative Medicine Strategies for Retina Repair . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 51 Linyang Yu, Vianney Delplace, Samantha L. Payne, and Molly S. Shoichet 4 Cell and Animal Models used for Retinal Stem Cell Research . . . . . . . 87 Michael J. Young and Jea Young Park 5 Limbal Stem Cells and the Treatment of Limbal Stem Cell Deficiency . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 123 Bruce R. Ksander, Markus H. Frank, and Natasha Y. Frank 6 Stem Cell Therapy and Regenerative Medicine in the Cornea . . . . . . . 149 Christopher D. McTiernan, Isabelle Brunette, and May Griffith 7 Clinical Applications of Limbal Stem Cells for Regenerative Medicine . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 173 Brian G. Ballios and Allan R. Slomovic 8 Retinal Ganglion Cell Replacement: A Bridge to the Brain . . . . . . . . . 193 Petr Baranov and Julia Oswald Index . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . . 207 vii Contributors Brian G. Ballios Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada Petr Baranov The Schepens Eye Research Institute of Massachusetts Eye and Ear, an affiliate of Harvard Medical School, Boston, MA, USA Gilbert Bernier Stem Cell and Developmental Biology Laboratory, Maisonneuve- Rosemont Hospital, Montréal, QC, Canada Department of Neuroscience, University of Montréal, Montréal, QC, Canada Department of Ophthalmology, University of Montréal, Montréal, QC, Canada Isabelle Brunette Maisonneuve-Rosemont Hospital Research Centre, Montreal, QC, Canada Department of Ophthalmology, University of Montreal, Montreal, QC, Canada Vianney Delplace Department of Chemical Engineering and Applied Chemistry, University of Toronto, Toronto, ON, Canada Clarisse M. Fligor Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, IN, USA Markus H. Frank Transplantation Research Center, Boston Children’s Hospital, Boston, MA, USA Department of Dermatology, Brigham and Women’s Hospital, Boston, MA, USA Natasha Y. Frank Department of Medicine, VA Boston Healthcare System, Harvard Medical School, Boston, MA, USA Division of Genetics, Brigham and Women’s Hospital, Boston, MA, USA May Griffith Maisonneuve-Rosemont Hospital Research Centre, Montreal, QC, Canada Department of Ophthalmology, University of Montreal, Montreal, QC, Canada ix x Contributors Kimberly T. Ho-A-Lim Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, IN, USA Bruce R. Ksander Schepens Eye Research Institute, Massachusetts Eye and Ear Infirmary, Boston, MA, USA Kirstin B. Langer Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, IN, USA Christopher D. McTiernan Maisonneuve-Rosemont Hospital Research Centre, Montreal, QC, Canada Department of Ophthalmology, University of Montreal, Montreal, QC, Canada Jason S. Meyer Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, IN, USA Department of Medical and Molecular Genetics, Indiana University, Indianapolis, IN, USA Stark Neurosciences Research Institute, Indiana University, Indianapolis, IN, USA Casey A. Miller Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, IN, USA Sarah K. Ohlemacher Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, IN, USA Julia Oswald The Schepens Eye Research Institute of Massachusetts Eye and Ear, an affiliate of Harvard Medical School, Boston, MA, USA Jea Young Park Department of Ophthalmology, Yale School of Medicine, New Haven, CT, USA Samantha L. Payne Department of Chemical Engineering and Applied Chemistry, University of Toronto, Toronto, ON, Canada Molly S. Shoichet Department of Chemical Engineering and Applied Chemistry, University of Toronto, Toronto, ON, Canada Allan R. Slomovic Department of Ophthalmology and Vision Sciences, University of Toronto, Toronto, ON, Canada Toronto General Hospital, University Health Network, Toronto, ON, Canada Department of Ophthalmology, Toronto Western Hospital, Toronto, ON, Canada Akshayalakshmi Sridhar Department of Biology, Indiana University Purdue University Indianapolis, Indianapolis, IN, USA Matthew Steinhart Medical Science Training Program, Indiana University, Bloomington, IN, USA Michael J. Young Schepens Eye Research Institute, Harvard Medical School, Boston, MA, USA Linyang Yu Department of Chemical Engineering and Applied Chemistry, University of Toronto, Toronto, ON, Canada Chapter 1 Photoreceptor Cell Replacement Therapy from Stem Cells Gilbert Bernier Abstract Macular degenerations, retinitis pigmentosa, and retinal dystrophies affect millions of people worldwide. In most cases, loss of visual function results from the death of photoreceptors, the specialized cells involved in photo- transduction. An innovative and efficient therapeutic solution for retinal degenera- tive diseases may be photoreceptor cell transplantation. Yet, the human eye contains about one hundred million photoreceptors, and cell replacement therapy would require at least a fraction of this, raising the issue of where to find an abundant source of healthy human photoreceptors to treat patients. Human pluripotent stem cells can be expanded quasi-indefinitely and differentiate into all cell types of the human body. Methods to direct the differentiation of human pluripotent stem cells into retinal cells and photoreceptors have been developed based on developmental biology principles. Here, we review the history and evolution of these methods, looking at two-dimensional and three-dimensional cell culture systems. We also analyze the current outcomes of photoreceptor cell transplantation therapy and explore the upcoming challenges for its clinical translation. Keywords Photoreceptor · Rod · Cone · Embryonic development · Neural induction · Stem cell · Pluripotent stem cell · Human · Retinal organoid · Retinal sheet · Retinal degeneration · Transplantation · Cell therapy G. Bernier (*) Stem Cell and Developmental Biology Laboratory, Maisonneuve-Rosemont Hospital, Montréal, QC, Canada Department of Neuroscience, University of Montréal, Montréal, QC, Canada Department of Ophthalmology, University of Montréal, Montréal, QC, Canada e-mail: [email protected] © Springer Nature Switzerland AG 2018 1 B. G. Ballios, M. J. Young (eds.), Regenerative Medicine and Stem Cell Therapy for the Eye, Fundamental Biomedical Technologies, https://doi.org/10.1007/978-3-319-98080-5_1
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