Cell-Based Therapy for Degenerative Retinal 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.

Explores why specific diseases are targeted for cell-based retinal therapy, including evidence that justifies optimism for the approach Discusses the challenges that must be managed in order to transition the technology from the laboratory into routine clinical practice Argues that de-risking can be achieved with preclinical experimentation in advance of human trials

Auteur
Dr. Zarbin graduated from the Johns Hopkins University School of Medicine (Medical Scientist Training Program, ) and completed resident and fellowship training (vitreoretinal surgery and medical retina) at the Wilmer Ophthalmological Institute. Dr. Zarbin is Chair of the Institute of Ophthalmology and Visual Science, Rutgers-New Jersey Medical School and Chief of Ophthalmology at University Hospital in Newark, N.J. He is a Professor of Ophthalmology and Neuroscience and is the Alfonse A. Cinotti, MD/Lions Eye Research Chair. Dr. Zarbin is co-Director of the Ocular Cell Transplantation Laboratory. Dr. Zarbin has published 221 peer-reviewed papers and editorials, 100 book chapters, one book on age-related macular degeneration, one book on stem cell therapy for degenerative retinal disease, and two books on the management of diabetic retinopathy.

Dr. Zarbin is a Vice Chair of the Scientific Advisory Board of the Foundation Fighting Blindness, Editor-in-Chief ofTranslational Vision Science and Technology, is a member of the editorial board of Ophthalmology, Ophthalmology Retina, and the Journal of Vitreoretinal Diseases, and is an ex officio member of the National Advisory Eye Council. He is also a member of the American Ophthalmological Society, Academia Ophthalmologica Internationalis, the Retina Society, the Macula Society, the Gonin Society, and the ASRS. Dr. Zarbin is an ARVO Fellow, Gold Level.

Dr. Mandeep S. Singh, M.D., Ph.D., graduated from the School of Medicine, National University of Singapore in 2001 and completed his residency in ophthalmology at the National University Hospital of Singapore. He obtained a Ph.D. in ophthalmology at the University of Oxford, United Kingdom in 2013. He completed his fellowship training in medical and surgical retina at the Oxford Eye Hospital and Moorfields Eye Hospital, United Kingdom. He is Assistant Professor of Ophthalmology in the Retina Division of the Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, USA. He has published 40 peer-reviewed papers and invited reviews, and five book chapters. He is a member of the Association for Research in Vision and Ophthalmology, the American Academy of Ophthalmology, the Club Jules Gonin and Euretina. He is a founding executive committee member of the Young Retinal Specialists (YOURS) initiative of Euretina. Dr. Singh received The Ruskell Medal in 2013 and the Fritz Tobler Foundation Award and the Johns Hopkins University School of Medicine Clinician Scientist Award in 2016. In the same year he received the European Vision Institute Publication of the Year recognition for the joint first authored paper entitled Emerging therapies for inherited retinal degeneration published in Science Translational Medicine. He received the Hartwell Foundation Individual Biomedical Research Award in 2017 and the Foundation Fighting Blindness Career Development Awardin 2018. He is the Principal Investigator for several observational and interventional clinical trials in retinal degenerative diseases, and leads a translational research laboratory focused on retinal stem cell therapy development.

Dr. Casaroli-Marano graduated from Faculty of Medical Sciences of Santa Casa (São Paulo, Brazil) and completed resident training at the Barraquer Institute (Barcelona, Spain) and further fellowship as Foreign Assistant at the Center Hospitalier National d'Ophtalmologie des Quinze-Vingt (Paris, France). He obtained a Master degree in Immunology, a PhD in Cell Biology and Pathology, and a post-PhD in Cell and Molecular Biology by the University of Barcelona. Currently, he is Professor of Ophthalmology at the School of Medicine in the University of Barcelona and Senior Consultant at the Hospital Clinic de Barcelona. He is Scientific Director at Barcelona Tissue Bank (BTB-BST) and researcher at the Institute of Biomedical Research (IIB-Sant Pau) and in the Spanish Network of Ophthalmological Research (OftaRed-RETICS ISCIII).

Dr. Casaroli-Marano is member of the expert committee of several national and international research agencies and is reviewer of several peer-review translational journals. He is a member of the Association for Research in Vision and Ophthalmology and co-editor with Dr. Zarbin of Cell-Based Therapy for Retinal Degenerative Disease. He is also Affiliate Professor at the Department of Ophthalmology and Visual Sciences, Escola Paulista de Medicina (UNIFESP, São Paulo, Brazil). He was co-founder of CellTec-UB, biotechnological spin-off at the University of Barcelona, and co-founder of Advanced In Vitro Cell Technologies-PCB (Advancell), a biotechnological and pharmaceutical initiative at the Scientific Park of Barcelona, where he served as scientific advisor, senior researcher and project leader.

Contenu

Advantages of the Eye as a Target Organ for Cell-Based Therapy in the Central Nervous System.- Proof of Principle: Preclinical Data on Retinal Cell Transplantation.- Clinical and Pathological Features of Selected Human Retinal Degenerative Diseases.- Degenerative Retinal Diseases: Cells sources for cell-based therapy.- Manufacturing of Clinical Grade Cellular Products Under Gmp Conditions.- Regulatory Requirements for Cell-Based Therapy for Degenerative Retinal Disease.- Biomaterials and Scaffolds for Cell Replacement Therapy.- Restoration of cone photoreceptor function in retinitis pigmentosa (RP) retinal cell-based therapy.- Cell Delivery: Surgical Approaches.- Diagnosis and Complementary Examinations.- Cell Therapy for Degenerative Retinal Disease: special focus on cell fusion-mediated regeneration.- Clinical Trials of Retinal Cell Therapy.- Future Directions.- Index.