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Autoimmune diseases are diverse and responsible for considerable morbidity. Their etiology remains largely unknown, and current therapy with anti-inflammatory drugs is prone to adverse effects, and rarely curative. New therapies with anti-cytokine antibodies or receptors are promising, but require frequent administration of expensive protein drugs. Gene Therapy of Autoimmune Diseases comprehensively reviews research in gene therapy for autoimmune diseases with viral or non-viral vectors. Gene therapy offers the possibility of long-term, continuous delivery of a wide variety of immunosuppressive, anti-inflammatory, or tolerance-inducing agents. Moreover, highly specific genetically modified cells can be produced. This book discusses the most promising avenues in this exciting new field.
The clinical management of autoimmune diseases has proven to be extremely difficult. Current therapies focus on trying to alleviate symptoms, but fail to correct the fundamental immune defects that lead to pathology. To achieve this goal, it is necessary to understand much of the biology of antigen presentation, lymphocyte activation and the effects of cytokines. The articles in this book provide an up-to-date review of current innovative therapies using both biologic and gene therapy for the treatment of selected autoimmune diseases. Therapeutical approaches discussed include oral tolerance, the use of anti-CD4 monoclonal antibodies, IL-10 and anti-TNFa antibodies, DNA vaccination, and gene therapy applied to organ-specific autoimmune disease. Although some of these techniques are still in their infancy, their potential efficacy has been demonstrated in several animal models of autoimmune disease, holding great promise for the future development of treatments. Written by recognized experts in the field, the chapters in this book illustrate the concept of technology transfer from bench to bedside and provide a valuable update for clinicians and scientists in clinical immunology.
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.
This book is devoted to innovative medicine, comprising the proceedings of the Uehara Memorial Foundation Symposium 2014. It remains extremely rare for the findings of basic research to be developed into clinical applications, and it takes a long time for the process to be achieved. The task of advancing the development of basic research into clinical reality lies with translational science, yet the field seems to struggle to find a way to move forward. To create innovative medical technology, many steps need to be taken: development and analysis of optimal animal models of human diseases, elucidation of genomic and epidemiological data, and establishment of “proof of concept”. There is also considerable demand for progress in drug research, new surgical procedures, and new clinical devices and equipment. While the original research target may be rare diseases, it is also important to apply those findings more broadly to common diseases. The book covers a wide range of topics and is organized into three complementary parts. The first part is basic research for innovative medicine, the second is translational research for innovative medicine, and the third is new technology for innovative medicine. This book helps to understand innovative medicine and to make progress in its realization.
A review of the field of cytokines in autoimmunity written by 22 researchers suggesting diverse applications, and building on the successes of recent rheumatoid arthritis clinical trials. The contributors present and review findings on arthritis, diabetes, multiple sclerosis, thyroid disease, Sjogren's syndrome, Lupus, scleroderma, and psoriasis. The final sections concentrate on laboratory and clinical concerns working with "knockout" mice, transgenic mice, and cytokine therapy. Distributed by Chapman and Hall. CiP shows the title as Role of cytokines..... Annotation copyright by Book News, Inc., Portland, OR
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.
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
The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were designed to encourage more pediatric studies of drugs used for children. The FDA asked the IOM to review aspects of pediatric studies and changes in product labeling that resulted from BPCA and PREA and their predecessor policies, as well as assess the incentives for pediatric studies of biologics and the extent to which biologics have been studied in children. The IOM committee concludes that these policies have helped provide clinicians who care for children with better information about the efficacy, safety, and appropriate prescribing of drugs. The IOM suggests that more can be done to increase knowledge about drugs used by children and thereby improve the clinical care, health, and well-being of the nation's children.
Written by experts in the dermatology field, this new fourth edition of Moderate-to-Severe Psoriasis discusses the current use of biologics and other pharmacologic and phototherapy treatments for moderate-to-severe psoriasis. Illustrated with high quality color figures, this standalone text emphasizes safe and effective treatments for the psoriasis
The field of pharmaceutical biotechnology is evolving rapidly. A whole new arsenal of protein pharmaceuticals is being produced by recombinant techniques for cancer, viral infections, cardiovascular and hereditary disorders, and other diseases. In addition, scientists are confronted with new technologies such as polymerase chain reactions, combinatorial chemistry and gene therapy. This introductory textbook provides extensive coverage of both the basic science and the applications of biotechnology-produced pharmaceuticals, with special emphasis on their clinical use. Pharmaceutical Biotechnology serves as a complete one-stop source for undergraduate pharmacists, and it is valuable for researchers and professionals in the pharmaceutical industry as well.