Clinical Trials In Retinitis Pigmentosa Treatment

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Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Author : National Academies of Sciences, Engineering, and Medicine
Publisher : National Academies Press
ISBN 13 : 0309672988
Total Pages : 127 pages
Book Rating : 4.3/5 (96 download)

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Book Synopsis Exploring Novel Clinical Trial Designs for Gene-Based Therapies by : National Academies of Sciences, Engineering, and Medicine

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.

Circadian and Visual Neuroscience

Author :
Publisher : Elsevier
ISBN 13 : 0323859445
Total Pages : 380 pages
Book Rating : 4.3/5 (238 download)

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Book Synopsis Circadian and Visual Neuroscience by :

Download or read book Circadian and Visual Neuroscience written by and published by Elsevier. This book was released on 2022-08-05 with total page 380 pages. Available in PDF, EPUB and Kindle. Book excerpt: Circadian and Visual Neuroscience, Volume 273 in the Methods in Enzymology series, highlights new advances in the field with this new volume presenting interesting chapters on topics including Optical set-ups, Psychophysics of Luminance and Color Vision, Psychophysics of non-visual photoreception PRC/IRC/DRC/Spectral Sensitivity, Circadian and visual photometry, Modelling (retina), Modelling (circadian), Techniques for examining vision at the cellular level, Advanced techniques for characterizing the world hyperspectrally, Circadian physiology in mice: Melanopsin, Circadian physiology in mice: Color and cones, Translational aspects of animal studies, Retinal clocks, Primate non-visual physiology, Light and mood in animal models, and much more. - Provides the authority and expertise of leading contributors from an international board of authors - Presents the latest release in Progress in Brain Research series - Updated release includes the latest information on Circadian and Visual Neuroscience

Autoimmune Neurology

Author :
Publisher : Elsevier
ISBN 13 : 0444634460
Total Pages : 569 pages
Book Rating : 4.4/5 (446 download)

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Book Synopsis Autoimmune Neurology by :

Download or read book Autoimmune Neurology written by and published by Elsevier. This book was released on 2016-03-11 with total page 569 pages. Available in PDF, EPUB and Kindle. Book excerpt: Autoimmune Neurology presents the latest information on autoimmune neurologic disease, the immune response to the body where organs run wild, causing the immune system to attack itself. Autoimmunity is a main element in numerous nervous system diseases and can target any structure within the central or peripheral nervous system. Over the past 20 years, significant advances in our understanding of the pathophysiology of autoimmune disorders, including the use of biomarkers has led to new diagnosis and treatment options. Neurologic conditions associated with autoimmune reactions include dementia, neuromuscular disease, epilepsy, sleep disorders, diabetes, and other common neurologic disorders and disease. This current tutorial-reference will be a must-have title for clinical neurologists, research neurologists, neuroscientists, and any medical professional working with autoimmune disease and disorders. - Includes comprehensive coverage of autoimmune neurology - Details the latest techniques for the study, diagnosis, and treatment of diseases and disorders, including dementia, neuromuscular disease, epilepsy, and sleep disorders - Presents a focused reference for clinical practitioners and the clinical neurology and neurology research communities

Retinal Degenerations

Author : Matthew M. LaVail
Publisher : Springer Science & Business Media
ISBN 13 : 1461500672
Total Pages : 458 pages
Book Rating : 4.4/5 (615 download)

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Book Synopsis Retinal Degenerations by : Matthew M. LaVail

Download or read book Retinal Degenerations written by Matthew M. LaVail and published by Springer Science & Business Media. This book was released on 2012-12-06 with total page 458 pages. Available in PDF, EPUB and Kindle. Book excerpt: The topics in this volume explore the etiology, cellular mechanisms, epidemiology, genetics, models and potential therapeutic measures for the blinding diseases of retinitis pigmentosa and age-related macular degeneration. Special focus is highlighted in the areas of Mechanisms of Photoreceptor Degeneration and Cell Death (extremely important because very little is known how or why photoreceptors die in these diseases, despite an abundance of genetic information), Age-Related Macular Degeneration (with several novel approaches to its analysis), Usher Syndrome (the most severe form of retinitis pigmentosa, which includes an early or congenital loss of hearing along with blindness), and Gene Therapy. In addition, the section on Basic Science Related to Retinal Degeneration is particularly strong with several laboratories reporting on new discoveries in the area of outer segment phagocytosis, a key component of photoreceptor-retinal pigment epithelial cell interactions in normal and degenerating retinas.

Atlas of Inherited Retinal Diseases

Author : Stephen H. Tsang
Publisher : Springer
ISBN 13 : 3319950460
Total Pages : 262 pages
Book Rating : 4.3/5 (199 download)

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Book Synopsis Atlas of Inherited Retinal Diseases by : Stephen H. Tsang

Download or read book Atlas of Inherited Retinal Diseases written by Stephen H. Tsang and published by Springer. This book was released on 2018-12-21 with total page 262 pages. Available in PDF, EPUB and Kindle. Book excerpt: This Atlas of Inherited Retinal Disorders provides a thorough overview of various inherited retinal dystrophies with emphasis on phenotype characteristics and how they relate to the most frequently encountered genes. It also meets the previously unmet needs of PhD students who will benefit from seeing the phenotypes of genes they work on and study. Further, because genetic-testing costs are quite high and spiraling higher, this Atlas will help geneticists familiarize themselves with the candidate gene approach to test patients’ genomes, enabling more cost-efficient testing. This invaluable atlas is organized into eight sections starting with an introduction to the basic knowledge on retinal imaging, followed by diseases listed according to inheritance pattern and disorders with extraocular manifestations grouped by defining features. This structure will be intuitive to clinicians and students studying inherited retinal disorders.

Retinal and Optic Nerve Stem Cell Surgery

Author : Jeffrey N. Weiss
Publisher : Springer Nature
ISBN 13 : 3030608506
Total Pages : 91 pages
Book Rating : 4.0/5 (36 download)

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Book Synopsis Retinal and Optic Nerve Stem Cell Surgery by : Jeffrey N. Weiss

Download or read book Retinal and Optic Nerve Stem Cell Surgery written by Jeffrey N. Weiss and published by Springer Nature. This book was released on 2020-11-19 with total page 91 pages. Available in PDF, EPUB and Kindle. Book excerpt: This is a concise how-to of successfully treating previously deemed "untreatable" retinal and optic nerve conditions with stem cell therapies. Based on the results of 11 years of experience treating approximately 600 patients, this text will serve as a template for others to develop similar stem cell based treatments in their specialty. Author, Jeffrey N. Weiss, MD is specifically poised to offer dependable insight on stem cell surgery for both the retina and the optic nerve, having performed the first retinal stem cell surgery in 2010, with Institutional Review Board (IRB) approved studies that are registered with the National Institute of Health (NIH), and listed on ClinicalTrials.gov. Featuring all original work from Dr. Jeffrey Weiss's specialization in stem cell ophthalmology, this book is primarily written for ophthalmologists, but structured so that specialists across disciplines can take the model presented and create their own studies and clinical trials using stem cell therapies.

Inherited Retinal Disease

Author : Hyeong-Gon Yu
Publisher : Springer Nature
ISBN 13 : 9811673373
Total Pages : 237 pages
Book Rating : 4.8/5 (116 download)

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Book Synopsis Inherited Retinal Disease by : Hyeong-Gon Yu

Download or read book Inherited Retinal Disease written by Hyeong-Gon Yu and published by Springer Nature. This book was released on 2022-03-11 with total page 237 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents the latest knowledge and expert guidance on all aspects of inherited retinal diseases, including molecular genetics, diagnosis, clinical features, general principles of treatment, novel treatment methods, and genetic counseling. Recent years have witnessed great advances in understanding of the genetic and cytological background of these diseases. Genetic analysis methods such as next generation sequencing have remarkably reduced the cost and time required for massive analysis of patients’ samples. Studies on gene therapy and stem cell therapy have been successfully carried out in animal models, and gene therapy is now available for Leber congenital amaurosis caused by RPE65 mutations. Against this background, Inherited Retinal Disease will be an invaluable up-to-date resource for ophthalmologists, medical students, and researchers in ocular inflammation. In addition to supplying essential information on each individual disorder, it features many interesting cases contributed by global leaders in the field as well as clinical photographs obtained with newer imaging techniques and numerous images of rare but clinically important diseases.

Fluorescence Lifetime Imaging Ophthalmoscopy

Author : Martin Zinkernagel
Publisher : Springer
ISBN 13 : 3030228789
Total Pages : 123 pages
Book Rating : 4.0/5 (32 download)

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Book Synopsis Fluorescence Lifetime Imaging Ophthalmoscopy by : Martin Zinkernagel

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 123 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.

Retinal Degenerative Diseases

Author : Matthew M. LaVail
Publisher : Springer Science & Business Media
ISBN 13 : 1461406315
Total Pages : 867 pages
Book Rating : 4.4/5 (614 download)

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Book Synopsis Retinal Degenerative Diseases by : Matthew M. LaVail

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.

Clinical Trials in Retinitis Pigmentosa Treatment

Author : Jeffrey N. Weiss
Publisher : Springer Nature
ISBN 13 : 3031587995
Total Pages : 281 pages
Book Rating : 4.0/5 (315 download)

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Book Synopsis Clinical Trials in Retinitis Pigmentosa Treatment by : Jeffrey N. Weiss

Download or read book Clinical Trials in Retinitis Pigmentosa Treatment written by Jeffrey N. Weiss and published by Springer Nature. This book was released on with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Handbook of Retinal OCT: Optical Coherence Tomography E-Book

Author : Jay S. Duker
Publisher : Elsevier Health Sciences
ISBN 13 : 0323757731
Total Pages : 274 pages
Book Rating : 4.3/5 (237 download)

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Book Synopsis Handbook of Retinal OCT: Optical Coherence Tomography E-Book by : Jay S. Duker

Download or read book Handbook of Retinal OCT: Optical Coherence Tomography E-Book written by Jay S. Duker and published by Elsevier Health Sciences. This book was released on 2021-07-31 with total page 274 pages. Available in PDF, EPUB and Kindle. Book excerpt: Arguably the most important ancillary test available to ophthalmologists worldwide, optical coherence tomography (OCT) has revolutionized the field, and now includes angiographic evaluations (OCTA) that provide vascular flow data without eye injection. Handbook of Retinal OCT is an easy-to-use, high-yield guide to both OCT and OCTA imaging for practitioners at any stage of their career. Highly templated, concise, and portable, this revised edition helps you master the latest imaging methods used to evaluate retinal disease, uveitis, and optic nerve disorders. - Helps all health professionals with an interest in OCT to better and more quickly interpret OCT imaging, offering quick, highly visual guidance for evaluating age-related macular degeneration, diabetic retinopathy, retinal vein occlusion, and much more. - Provides quick answers with bulleted, templated chapters, each focused on one specific diagnosis or group of diagnoses with a particular OCT appearance. - Demonstrates how the full spectrum of diseases presents through approximately 400 illustrations, including the highest-quality spectral-domain OCT images available and more than 50 new OCTA images. - Includes five new chapters covering optic nerve disease with retinal findings, pachychoroid diseases, paracentral acute middle maculopathy (PAMM), auto-immune retinopathies, and primary uveal lymphoma. - Offers clear visual guidance on image patterns with multiple arrows and labels throughout to highlight key details of each disease.

Macular Dystrophies

Author : Giuseppe Querques
Publisher : Springer
ISBN 13 : 3319266217
Total Pages : 128 pages
Book Rating : 4.3/5 (192 download)

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Book Synopsis Macular Dystrophies by : Giuseppe Querques

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.

Precision Medicine, CRISPR, and Genome Engineering

Author : Stephen H. Tsang
Publisher : Springer
ISBN 13 : 3319639048
Total Pages : 180 pages
Book Rating : 4.3/5 (196 download)

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Book Synopsis Precision Medicine, CRISPR, and Genome Engineering by : Stephen H. Tsang

Download or read book Precision Medicine, CRISPR, and Genome Engineering written by Stephen H. Tsang and published by Springer. This book was released on 2017-11-11 with total page 180 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents descriptive overviews of gene editing strategies across multiple species while also offering in-depth insight on complex cases of application in the field of tissue engineering and regenerative medicine. Chapters feature contributions from leaders in stem cell therapy and biology, providing a comprehensive view of the application of gene therapy in numerous fields with an emphasis on ophthalmology, stem cells, and agriculture. The book also highlights recent major technological advances, including ZFN, TALEN, and CRISPR. Precision Medicine, CRISPR, and Genome Engineering is part of the highly successful Advances in Experimental Medicine and Biology series. It is an indispensable resource for researchers and students in genetics as well as clinicians.

Principles and Practice of Clinical Electrophysiology of Vision, second edition

Author : John R. Heckenlively
Publisher : MIT Press
ISBN 13 : 0262083469
Total Pages : 1033 pages
Book Rating : 4.2/5 (62 download)

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Book Synopsis Principles and Practice of Clinical Electrophysiology of Vision, second edition by : John R. Heckenlively

Download or read book Principles and Practice of Clinical Electrophysiology of Vision, second edition written by John R. Heckenlively and published by MIT Press. This book was released on 2006-04-07 with total page 1033 pages. Available in PDF, EPUB and Kindle. Book excerpt: The long-awaited second edition of an authoritative reference on electrophysiologic vision testing, including detailed information on techniques and problems, basic physiology and anatomy, theoretical concepts, and clinical findings; with extensive new material. This authoritative text is the only comprehensive reference available on electrophysiologic vision testing, offering both practical information on techniques and problems as well as basic physiology and anatomy, theoretical concepts, and clinical correlations. The second edition, of the widely used text, offers extensive new material and updated information: 65 of the 84 chapters are completely new, with the changes reflecting recent advances in the field. The book will continue to be an essential resource for practitioners and scholars from a range of disciplines within vision science. The contributions not only cover new information—important material that is likely to become more important in the next decade—but also offer a long-range perspective on the field and its remarkable development in the last century. After discussing the history and background of clinical electrophysiology, the book introduces the anatomy of the retina and principles of cell biology in the visual pathways at the molecular, physiological, and biochemical levels. It relates these new findings to the techniques and interpretations of clinical tests, including the electro-oculogram (EOG), electroretinogram (ERG), and visual evoked potentials (VEP), which are discussed in detail, as are equipment, data acquisition and analysis, principles and protocols for clinical testing, diseases and dysfunction, and animal testing. Notable additions for this edition include chapters on the origin of electroretinogram waveforms, multifocal techniques, testing in standard laboratory animals, recent advances in analysis of abnormalities in disease, and the applications of these techniques to the study of genetic abnormalities.

Gene Transfer and the Ethics of First-in-Human Research

Author : Jonathan Kimmelman
Publisher : Cambridge University Press
ISBN 13 : 0521690846
Total Pages : 219 pages
Book Rating : 4.5/5 (216 download)

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Book Synopsis Gene Transfer and the Ethics of First-in-Human Research by : Jonathan Kimmelman

Download or read book Gene Transfer and the Ethics of First-in-Human Research written by Jonathan Kimmelman and published by Cambridge University Press. This book was released on 2010 with total page 219 pages. Available in PDF, EPUB and Kindle. Book excerpt: Examines the ethical and policy dimensions of testing novel medical interventions in human beings for the first time.

Recent Advances In Retinal Degeneration

Author : Robert E. Anderson
Publisher : Springer Science & Business Media
ISBN 13 : 0387749047
Total Pages : 422 pages
Book Rating : 4.3/5 (877 download)

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Book Synopsis Recent Advances In Retinal Degeneration by : Robert E. Anderson

Download or read book Recent Advances In Retinal Degeneration written by Robert E. Anderson and published by Springer Science & Business Media. This book was released on 2007-12-03 with total page 422 pages. Available in PDF, EPUB and Kindle. Book excerpt: The product of perhaps the most important research meeting in the field, this essential text outlines all the latest research in retinal degeneration. Culled from the proceedings of the International Symposium on the subject, the topics in this volume explore the etiology, cellular mechanisms, epidemiology, models and potential therapeutic measures for the blinding diseases of retinitis pigmentosa and age-related macular degeneration. A must-read for researchers in the field.

Cell-Based Therapy for Degenerative Retinal Disease

Author : Marco A. Zarbin
Publisher : Springer
ISBN 13 : 3030052222
Total Pages : 286 pages
Book Rating : 4.0/5 (3 download)

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Book Synopsis Cell-Based Therapy for Degenerative Retinal Disease by : Marco A. Zarbin

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 286 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.