Cellular Therapies For Retinal Disease

This book familiarizes the reader with the current landscape of cell-based therapies for the treatment of retinal disease, including diseases that affect the choriocapillaris, retinal pigment epithelium, photoreceptors, and retinal ganglion cells. Instead of utilizing a disease-centric approach to the topic, this book—edited by two world-renowned stem cell scientists—focuses on strategies for developing and transplanting the cells. This includes the creation of replacement cells, cell-based neuroprotection, and in vitro disease modeling and testing. The final chapters briefly review parallel approaches that do not directly utilize cellular transplantation. The use of cellular transplantation to treat retinal disease has recently become a viable and exciting therapeutic approach. The visibility of the retina and its laminar cellular architecture render it an ideal organ for the development of surgically delivered cellular therapies. Having an in-depth understanding of the current state of cell therapy for the eye is an essential first step toward utilizing similar approaches in other organs. Ophthalmologists, translational clinician-scientists, stem cell scientists, and researchers interested in eye disease will find Cellular Therapies for Retinal Disease: A Strategic Approach essential reading and it is also suitable for workshops or courses at the undergraduate or Ph.D. level.

In this volume, some of the leading authorities present their exploration of applications of stem cell therapy to the treatment of major causes of blindness, including degenerative diseases and glaucoma. The diagnostic approach to patients, general concepts of cell-based therapy, immunological considerations, approaches to cell delivery (including engineered scaffolds), combined cell and gene therapy, nanomedicine applications to cell therapy and regulatory issues pertaining to manufacture and production are all considered in detail. The book serves as an excellent introduction to a field that is now entering early-stage clinical trials and promises to operate at the leading edge of regenerative medicine. Retina specialists, general ophthalmologists as well as researchers will find here a wealth of information on the translational aspects of cell-based therapies. Further, business executives and students interested in understanding the potential applications of stem cell therapy to retinal degenerative disease and glaucoma will also find this book informative reading.

This book discusses why specific diseases are being targeted for cell-based retinal therapy, what evidence exists that justifies optimism for this approach, and what challenges must be managed in order to bring this technology from the laboratory into routine clinical practice. There are a number of unanswered questions (e.g., surgical approach to cell delivery, management of immune response, optimum cell type to transplant) that very likely are not going to be answered until human trials are undertaken, but there is a certain amount of “de-risking” that can be done with preclinical experimentation. This book is essential reading for scientists, clinicians, and advanced students in stem cell research, cell biology, and ophthalmology.

This book discusses applications of pluripotent stem cells to study eye disease in vitro and to create novel therapies for degenerative eye diseases. Chapters are contributed by experts in the field and cover such topics as the use of pluripotent stem cells in 2D and 3D engineering of ocular tissues for disease modelling and drug testing as well as approaches to replace degenerated RPE and photoreceptors in macular degeneration and retinitis pigmentosa. Pluripotent Stem Cells in Eye Disease Therapy presents a comprehensive discussion of basic science and clinical applications and is an indispensable resource for everyone from advanced graduate students to advanced professionals who want to learn about the potential of stem cell biology and its role in the field of retinal diseases.

This book provides an overview of the types, sources, and applications of stem cells in regenerating various ocular tissues, with a perspective on both potential applications of stem cells and possible challenges. The scope of the chapters include both preclinical and clinical applications, including stem cell-derived therapies based on endogenous tissue repair; stem cell transplantation and cell replacement therapy; gene therapy; and in vitro disease modelling. Additionally, the volume presents applications in both anterior and posterior ocular disease, with a particular focus on diseases of the ocular surface, cornea, limbus, and retina, including inherited retinal dystrophies as well as acquired diseases, such as age-related macular degeneration. Regenerative Medicine and Stem Cell Therapy for the Eye is an ideal book for advanced researchers in stem cell and ocular biology as well as clinical ophthalmologists, and will be of interest to readers with backgrounds in developmental biology and bioengineering. This book also Skillfully reviews cutting-edge advances in stem cell biology as applied to regenerative medicine and ocular disease Provides expert viewpoints on key hurdles and challenges to successful implementation of stem cell-derived therapies in the clinical domain Offers a multi-disciplinary, broad understanding of cell-based therapies for ocular diseases by incorporating perspectives from biomedical scientists, physicians, and engineers Examines the connection between cell therapy and gene editing, in particular relation to ocular disease

This book discusses why specific diseases are being targeted for cell-based retinal therapy, what evidence exists that justifies optimism for this approach, and what challenges must be managed in order to bring this technology from the laboratory into routine clinical practice. There are a number of unanswered questions (e.g., surgical approach to cell delivery, management of immune response, optimum cell type to transplant) that very likely are not going to be answered until human trials are undertaken, but there is a certain amount of "de-risking" that can be done with preclinical experimentation. This book is essential reading for scientists, clinicians, and advanced students in stem cell research, cell biology, and ophthalmology.

There are more than 300 genes that have been identified which carry mutations that cause various forms of retinal dysfunction and degeneration, making the study of retinal diseases a subject of high relevance. In this compendium of original and review articles, many of the diseases and pathways associated with disorders of the retina are examined using animal models, to provide the reader with a good overview of current retinal research. Within this volume, you will find research reports on many of the most prominent retinal disorders, such as diabetic retinopathy (DR), age-related macular degeneration (AMD), choroidal neovascularization (CNV), and retinitis pigmentosa (RP). We hope that the work presented here will stimulate new ideas and lead to effective treatments for retinal diseases.

Contains the proceedings of the XVI International Symposium on Retinal Degeneration (RD2014), to be held July 13-18, 2014 at the Asilomar Conference Center in Pacific Grove, California. A majority of those who will speak and present posters at the meeting will contribute to this volume. The Symposium addresses the blinding diseases of inherited retinal degenerations, which have no effective treatments and age-related macular degeneration, which has no cures, despite the fact that it is an epidemic among the elderly, with 1 in 3-4 affected by the age of 75. The RD2014 Symposium will focus on the exciting new developments aimed at understanding these diseases and providing therapies for them. The volume will present representative state-of-the-art research in almost all areas of retinal degenerations, ranging from cytopathologic, physiologic, diagnostic and clinical aspects; animal models; mechanisms of cell death; molecular genetics; and developing potential therapeutic measures such as gene therapy and neuroprotective agents for potential pharmaceutical therapy; and several sight restoration approaches, including optogenetics. While advances in these areas of retinal degenerations will be included, several new topics either were in their infancy or did not exist at the time of the last RD Symposium, RD2012. These include many new developments in sight restoration using optogenetics, retinal or RPE cell transplantation, stem cell approaches and visual prosthetic devices. In addition, major advances will be presented in other basic mechanisms in age-related macular degeneration, several new aspects of gene and antioxidant therapy and revolutionary new imaging and functional testing that will have a huge impact on the diagnosis and following the course of retinal degenerations, as well as to provide new quantitative endpoints for clinical trials. The retina is an approachable part of the central nervous system (CNS), and there is a major interest in neuroprotective and gene therapy for CNS diseases and neurodegenerations, in general. It should be noted that with successful and exciting initial clinical trials in neuroprotective and gene therapy, including the restoration of sight in blind children, the retinal degeneration therapies are leading the way towards new therapeutic measures for neurodegenerations of the CNS. Many of the successes recently reported in these areas of retinal degeneration sprang from collaborations established at previous RD Symposia, and many of those will be reported at the RD2014 meeting and included.