Download or read book Gene and Cell Based Treatment Strategies for the Eye written by P. Elizabeth Rakoczy and published by Springer. This book was released on 2015-01-17 with total page 95 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this book, leading experts provide detailed descriptions of the exciting treatments that are expected to become part of the ophthalmologist’s arsenal within the next 10–20 years. The treatments discussed relate to a wide variety of conditions, including macular degeneration, Leber’s congenital amaurosis, retinitis pigmentosa, choroideremia, and retinoschisis. The authors explain clearly how different gene and cell therapies work and provide first-hand accounts of the difficulties that they have faced in bringing these technologies to clinical trial, such as issues relating to funding and ownership. Results achieved to date are presented, and the further steps required before the treatment in question can become a routine option are considered. Gene- and Cell-Based Treatment Strategies for the Eye is unique in showing the organic development of cutting-edge science into potential treatments for eye disease without compromising on accurate reporting of scientific facts. It will persuade the average practitioner or researcher – whether ophthalmologist, health worker, or scientist – that change is indeed coming and is not just a hollow promise of the tabloid media.

Download or read book Cell Based Therapy for Retinal Degenerative Disease written by R.P. Casaroli-Marano and published by Karger Medical and Scientific Publishers. This book was released on 2014-04-22 with total page 220 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Cellular Therapies for Retinal Disease written by Steven D. Schwartz and published by Springer. This book was released on 2017-06-20 with total page 149 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Ocular Neuroprotection written by Leonard A. Levin and published by CRC Press. This book was released on 2003-07-15 with total page 343 pages. Available in PDF, EPUB and Kindle. Book excerpt: Many retinal and optic nerve disorders have no effective therapy, or are treated incompletely and with considerable side effects. Recent advances have suggested the significant benefits associated with neuroprotection - that is, when treatment strategies are directed to photoreceptors, retinal ganglion cells, or other neural targets. Enter Ocular N

Download or read book Gene and Cell Based Treatment Strategies for the Eye written by Elizabeth P. Rakoczy and published by Springer. This book was released on 2015-01-13 with total page 101 pages. Available in PDF, EPUB and Kindle. Book excerpt: In this book, leading experts provide detailed descriptions of the exciting treatments that are expected to become part of the ophthalmologist’s arsenal within the next 10–20 years. The treatments discussed relate to a wide variety of conditions, including macular degeneration, Leber’s congenital amaurosis, retinitis pigmentosa, choroideremia, and retinoschisis. The authors explain clearly how different gene and cell therapies work and provide first-hand accounts of the difficulties that they have faced in bringing these technologies to clinical trial, such as issues relating to funding and ownership. Results achieved to date are presented, and the further steps required before the treatment in question can become a routine option are considered. Gene- and Cell-Based Treatment Strategies for the Eye is unique in showing the organic development of cutting-edge science into potential treatments for eye disease without compromising on accurate reporting of scientific facts. It will persuade the average practitioner or researcher – whether ophthalmologist, health worker, or scientist – that change is indeed coming and is not just a hollow promise of the tabloid media.

Download or read book Cell Based Therapy for Degenerative Retinal Disease written by Marco A. Zarbin and published by Springer. This book was released on 2019-03-01 with total page 280 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Pluripotent Stem Cells in Eye Disease Therapy written by Kapil Bharti and published by Springer Nature. This book was released on 2019-10-25 with total page 201 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Exploring Novel Clinical Trial Designs for Gene Based Therapies written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-08-27 with total page 127 pages. Available in PDF, EPUB and Kindle. Book excerpt: Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

Download or read book Retinal Degenerative Diseases written by Matthew M. LaVail and published by Springer Science & Business Media. This book was released on 2011-12-21 with total page 867 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book will contain the proceedings of the XIV International Symposium on Retinal Degeneration (RD2010), held July 13-17, 2010, in Mont-Tremblant, Quebec, Canada. 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; candidate genes, cloning, mapping and other aspects of molecular genetics; and developing potential therapeutic measures such as gene therapy and neuroprotective agents for potential pharmaceutical therapy.

Download or read book Advances in Vision Research Volume III written by Gyan Prakash and published by Springer Nature. This book was released on 2021-02-18 with total page 478 pages. Available in PDF, EPUB and Kindle. Book excerpt: This third volume, with three supporting editors, broadens its focus on genetic eye research from the Asian to the global scale. New efforts and a new awareness have sparked important discussions on genetic eye research, and new plans are being implemented to identify the genes responsible for numerous eye diseases. The book introduces the latest findings on genetics in eye diseases, gene therapy, and genome-wide association analysis, and the efforts of the Global Eye Genetic Consortium (GEGC). The book’s editors have been instrumental in developing strategies for discovering the new genes involved in many eye diseases. All chapters were written by leading researchers working on eye genetics from the fields of Human Genetics, Ophthalmology, Molecular Biology, Biochemistry, Sensory Sciences, and Clinical Research. Advances in Vision Research, Volume III is a major resource for all researchers, clinicians, clinical researchers, and allied eye health professionals with an interest in eye diseases around the globe.

Download or read book Treatment of Non infectious Uveitis written by Phoebe Lin and published by Springer Nature. This book was released on 2019-09-12 with total page 205 pages. Available in PDF, EPUB and Kindle. Book excerpt: This unique, comprehensive book provides a much-needed reference on the treatment and management of non-infectious uveitis. Carefully designed, Treatment of Non-infectious Uveitis is the first book of its kind to provide an in-depth, clinically-relevant, expert-driven resource for ophthalmologists focusing on modalities of uveitis treatment, their mechanism of action, dosing, and side effects. Each chapter provides an introduction, mechanism of action, indication, dosage, side effects, and efficacy summaries from clinical trials and other published literature. Topics range from topical treatment, to locally administered therapy including drug-releasing implants, to systemic immunosuppressive treatments both tried and new, as well as surgical management, with each chapter highlighting important practice pearls as well as easy-reference dosing tables, side effects, and lab monitoring pertinent to the agents discussed. The book concludes with a discussion of novel approaches to the treatment of non-infectious uveitis, and special considerations when treating uveitis in the pediatric patient. The majority of patients with non-infectious uveitis are treated by comprehensive ophthalmologists, many of whom are less familiar with established treatment guidelines outlining the role of corticosteroids and immunomodulatory therapy. While the non-specialist, resident, or fellow is sure to benefit from this one-stop guide to uveitis treatment, retina and uveitis specialists alike will also appreciate the practice tips and thorough coverage of this expertly-written reference. Treatment of Non-infectious Uveitis is the ideal reference for all ophthalmologists who seek to improve their understanding of the causes of uveitis and learn how to best treat this condition.

Download or read book Fluorescence Lifetime Imaging Ophthalmoscopy written by Martin Zinkernagel and published by Springer. This book was released on 2019-07-29 with total page 121 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book focuses on the emerging non-invasive imaging technique of Fluorescence Lifetime Imaging Ophthalmoscopy (FLIO). FLIO reveals unique information on retinal diseases, ranging from age-related macular degeneration and vascular diseases to hereditary retinal dystrophies. Fluorescence lifetimes enable the evaluation of disease progression before irreversible structural changes occur. The image acquisition is suitable for diagnostic purposes and follow-up examinations to investigate the natural course of disease, and to monitor the effects of possible therapies. This book fills the gap between available literature and gives state-of-the-art guidance on the principles of the FLIO technique, image acquisition, and data analysis. Written by a team of expert leaders within this field, this book will be relevant for scientists and clinicians with an interest in ophthalmoscopy.

Download or read book Retinal Dystrophies written by Novartis Foundation and published by Wiley. This book was released on 2004-01-16 with total page 226 pages. Available in PDF, EPUB and Kindle. Book excerpt: Retinal dystrophies are the major causes of incurable blindness in the Western world. Our insight into their aetiology has improved remarkably over the past decade and a number of key genes have been identified. Together with a more detailed understanding of disease processes, this knowledge is stimulating new approaches to therapeutic strategies involving gene therapy, growth factors and retinal cell transplantation. Molecular genetic studies have provided detailed information on the pathogenesis of retinal dystrophies. An important proof of principle that gene therapy holds great promise for the treatment of these conditions was demonstrated in the rds mouse: introduction of a functional copy of the peripherin gene subretinally resulted in complete rescue of rod outer segment structure. Novel approaches are being developed based on the manipulation of biochemical pathways that previously were not considered relevant to these diseases. For example, renewed interest in retinal dystrophy pathogenesis led to the successful use of high dose vitamin A treatment in Sorsby fundus dystrophy. This important new book covers all aspects of retinal dystrophies from the molecular and developmental biology of these disorders to possible therapeutic approaches, with special reference to gene therapy. Specific chapters deal with the molecular genetics of gene therapies, clinical genetic studies, molecular and cellular mechanisms of the development of the disease, functional genomics of retinal diseases, animal models of retinal dystrophies, and finally with studies on gene therapeutic approaches to correcting the disorder. With contributions by many of the leading researchers worldwide, this book is likely to be an important milestone in this rapidly developing field.

Download or read book Advanced Therapy Medicinal Products for Eye Diseases Goals and Challenges written by Yolanda Diebold and published by . This book was released on 2021 with total page 207 pages. Available in PDF, EPUB and Kindle. Book excerpt: The concept of advanced therapy medicinal products (ATMPs) encompasses novel kinds of medicines for human use that are based on genes, cells or tissues. These intend to offer not only regeneration, but complete functional recovery of diseased tissues and organs using different strategies. Gene therapy, cell therapy and tissue engineering are the main areas in which promising advanced therapies are emerging. The eye is a very complex organ whose main structures, the cornea and the retina, play a pivotal role in maintaining normal vision, as severe alterations in these tissues can lead to blindness. Ocular tissues are starting to benefit from ATMPs by fighting against the enormous complexity and devastating potential of many ocular diseases. However, developments arising from this field of work face important challenges related to vectors to deliver drugs and genetic material to target tissues, suitable biomaterials to prepare cell scaffolds and cell stemness, among others--not to mention the complicated legislation around ATMPs, the complexity in production and quality control and the absence of standardized protocols.The purpose of this Special Issue is to serve as an overview of the current progress in the application of cell and gene therapies, as well as tissue engineering to restore functionality in diseased ocular structures, and the challenges linked to reaching patients.

Download or read book Macular Dystrophies written by Giuseppe Querques and published by Springer. This book was released on 2016-02-15 with total page 128 pages. Available in PDF, EPUB and Kindle. Book excerpt: ​This book provides the ophthalmologist with the most recently available data on the macular dystrophies, a group of many different inherited or sporadic eye conditions linked by a problem with photoreceptors or other structures of the central retina. Internationally recognized experts in the field present the latest evidence and discuss their own personal experiences with regard to each of the principal dystrophies as well as some very rare entities. Topics covered include molecular biology, state-of-the-art diagnostic techniques, and the newest treatment options, including still experimental therapies. Attention is also devoted to a range of issues that continue to be debated. The editors have taken care to ensure that chapters are of a uniformly high standard while not sacrificing the originality of the individual authors. Macular Dystrophies will fully acquaint the reader with both the latest research findings and the current and emerging approaches to diagnosis and treatment.

Download or read book Regulatory Aspects of Gene Therapy and Cell Therapy Products written by Maria Cristina Galli and published by Springer. This book was released on 2015-09-15 with total page 235 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Download or read book A Guide to Human Gene Therapy written by Roland W. Herzog and published by World Scientific. This book was released on 2010 with total page 415 pages. Available in PDF, EPUB and Kindle. Book excerpt: 1. Non-viral gene therapy / Sean M. Sullivan -- 2. Adenoviral vectors / Stuart A. Nicklin and Andrew H. Baker -- 3. Retroviral vectors and integration analysis / Cynthia C. Bartholomae [und weitere] -- 4. Lentiviral vectors / Janka Matrai, Marinee K.L. Chuah and Thierry VandenDriessche -- 5. Herpes simplex virus vectors / William F. Goins [und weitere] -- 6. Adeno-Associated Viral (AAV) vectors / Nicholas Muzyczka -- 7. Regulatory RNA in gene therapy / Alfred. S. Lewin -- 8. DNA integrating vectors (Transposon, Integrase) / Lauren E. Woodard and Michele P. Calos -- 9. Homologous recombination and targeted gene modification for gene therapy / Matthew Porteus -- 10. Gene switches for pre-clinical studies in gene therapy / Caroline Le Guiner [und weitere] -- 11. Gene therapy for central nervous system disorders / Deborah Young and Patricia A. Lawlor -- 12. Gene therapy of hemoglobinopathies / Angela E. Rivers and Arun Srivastava -- 13. Gene therapy for primary immunodeficiencies / Aisha Sauer, Barbara Cassani and Alessandro Aiuti -- 14. Gene therapy for hemophilia / David Markusic, Babak Moghimi and Roland Herzog -- 15. Gene therapy for obesity and diabetes / Sergei Zolotukhin and Clive H. Wasserfall -- 16. Gene therapy for Duchenne muscular dystrophy / Takashi Okada and Shin'ichi Takeda -- 17. Cancer gene therapy / Kirsten A.K. Weigel-Van Aken -- 18. Gene therapy for autoimmune disorders / Daniel F. Gaddy, Melanie A. Ruffner and Paul D. Robbins -- 19. Gene therapy for inherited metabolic storage diseases / Cathryn Mah -- 20. Retinal diseases / Shannon E. Boye, Sanford L. Boye and William W. Hauswirth -- 21. A brief guide to gene therapy treatments for pulmonary diseases / Ashley T. Martino, Christian Mueller and Terence R. Flotte -- 22. Cardiovascular disease / Darin J. Falk, Cathryn S. Mah and Barry J. Byrne