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Author: Jeffrey Laurence Publisher: Academic Press ISBN: 0128005645 Category : Science Languages : en Pages : 346
Book Description
Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials
Author: Jeffrey Laurence Publisher: Academic Press ISBN: 0128005645 Category : Science Languages : en Pages : 346
Book Description
Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme. Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details specific progress and barriers for current translational use Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials
Author: Jeffrey Laurence Publisher: Academic Press ISBN: 0128005521 Category : Technology & Engineering Languages : en Pages : 356
Book Description
Translating Regenerative Medicine to the Clinic reviews the current methodological tools and experimental approaches used by leading translational researchers, discussing the uses of regenerative medicine for different disease treatment areas, including cardiovascular disease, muscle regeneration, and regeneration of the bone and skin. Pedagogically, the book concentrates on the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field. It promotes cross-disciplinary communication between the sub-specialties of medicine, but remains unified in theme by emphasizing recent innovations, critical barriers to progress, the new tools that are being used to overcome them, and specific areas of research that require additional study to advance the field as a whole. Volumes in the series include Translating Gene Therapy to the Clinic, Translating Regenerative Medicine to the Clinic, Translating MicroRNAs to the Clinic, Translating Biomarkers to the Clinic, and Translating Epigenetics to the Clinic. Encompasses the latest innovations and tools being used to develop regenerative medicine in the lab and clinic Covers the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field Contains extensive pedagogical updates aiming to improve the education of translational researchers in this field Provides a transdisciplinary approach that supports cross-fertilization between different sub-specialties of medicine
Author: Kam M Hui Publisher: World Scientific ISBN: 9814502383 Category : Science Languages : en Pages : 289
Book Description
With advances in our understanding of the molecular biology of human diseases and the development of efficient gene transfer techniques, the treatment of such diseases as cancer and infectious disease using gene therapy has progressed from a distant prospect to a distinct possibility in a very short time. The development of gene transfer methods which are suitable for different forms of therapy has been a major topic of research over the past several years. A common goal of this research has been to achieve the efficient delivery of genes into cells. The successful implementation of gene transfer as a cure for diseases, however, will continue to require the translation of preclinical studies in gene therapy into effective clinical protocols. This volume outlines the latest developments in cancer treatment using various gene delivery systems, which include cytokine gene transfer, the delivery of anti-ras DNA by retroviral vector and the injection of allogeneic HLA DNA via liposomes. Several of these molecular approaches have recently been approved by the US FDA as human clinical trial protocols in order to assess their therapeutic efficiency and safety for cancer treatment. Further developments in recombinant DNA technology within this field should ultimately lead to dramatic improvements in the practice of medicine.
Author: Kelly K. Hunt Publisher: Springer Science & Business Media ISBN: 159745222X Category : Medical Languages : en Pages : 469
Book Description
The three sections of this volume present currently available cancer gene therapy techniques. Part I describes the various aspects of gene delivery. In Part II, the contributors discuss strategies and targets for the treatment of cancer. Finally, in Part III, experts discuss the difficulties inherent in bringing gene therapy treatment for cancer to the clinic. This book will prove valuable as the volume of preclinical and clinical data continues to increase.
Author: National Academies of Sciences, Engineering, and Medicine Publisher: National Academies Press ISBN: 0309495849 Category : Medical Languages : en Pages : 93
Book Description
On April 23 and 24, 2019 the Forum on Neuroscience and Nervous System Disorders convened a workshop titled "Advancing Gene-Targeted Therapies for Central Nervous System Disorders" in Washington, DC. This public workshop brought together experts and key stakeholders from academia, government, industry, philanthropic foundations, and disease/patient-focused nonprofit organizations to explore approaches for advancing the development of gene-targeted therapies for central nervous system (CNS) disorders, and implications of developing these therapies. Participants explored lessons learned from both successful and unsuccessful clinical development programs; new knowledge about the genetic underpinnings of brain disorders; the current status and future potential of gene-targeted therapies for CNS disorders; challenges and potential solutions for translating preclinical findings to approved therapies; and patient and caregiver perspectives. They also discussed what will be needed to develop these therapies for common disorders such as Alzheimer's and Parkinson's disease, as well as neuropsychiatric and neurodevelopmental disorders such as schizophrenia and autism. The workshop included approaches that target both DNA and RNA, as well as gene products using viral vectors, antisense oligonucleotides, and RNA interference. This publication summarizes the presentations and discussion of the workshop.
Author: Publisher: Academic Press ISBN: 0128024704 Category : Science Languages : en Pages : 296
Book Description
The field of genetics is rapidly evolving, and new medical breakthroughs are occurring as a result of advances in our knowledge of genetics. Advances in Genetics continually publishes important reviews of the broadest interest to geneticists and their colleagues in affiliated disciplines. Includes methods for testing with ethical, legal, and social implications Critically analyzes future directions Written and edited by recognized leaders in the field
Author: Michael G. Kaplitt Publisher: Elsevier ISBN: 9780080454375 Category : Science Languages : en Pages : 370
Book Description
Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis. * Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells * Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis * Includes "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use
Author: Odile Cohen-Haguenauer Publisher: EDK Editions ISBN: 2842542371 Category : Medical Languages : en Pages : 589
Book Description
This book provides a 2012 reference for state-of-the-art gene transfer technology and the different aspects of its clinical translation with a focus on European-based initiatives. As examples of successful outcomes, recent clinical trials are presented together with Ethical, Safety and Regulatory issues, which are discussed. The broad range of various technologies is covered whether addressing direct in vivo gene transfer like with AAV, Adeno or non-viral vectors or ex-vivo genetically engineered cells including induced pluripotent stem cells (iPS) with integrating vectors such as retrovirus, lentivirus or transposon-derived systems. The critical path to clinical implementation is covered in the second part describing currently available tools - such as molecular imaging, ex-vivo organ cultures and high-throughput technologies used for evaluation of criteria towards a go-or-no go decision to move to the clinic; in addition, utmost salient biosafety and immunotoxicology aspects are discussed. This book is ideal for postgraduates, undergraduates, scientists, clinicians, regulators and patients' advocacy groups looking for states-of-the-art information as well as emerging prospects - including gene targeting and homologous recombination - in gene transfer intended for clinical translation. Dr Odile Cohen-Haguenauer is the Coordinator of Clingene-NoE, the European Network for the Advancement of Clinical Gene Transfer and Therapy.
Author: Nicholas M Boulis Publisher: Academic Press ISBN: 0128025247 Category : Psychology Languages : en Pages : 336
Book Description
Molecular and Cellular Therapies for Motor Neuron Diseases discusses the basics of the diseases, also covering advances in research and clinical trials. The book provides a resource for students that will help them learn the basics in a detailed manner that is required for scientists and clinicians. Users will find a comprehensive overview of the background of Amyotrophic Lateral Sclerosis (ALS/Lou Gehrig’s Disease) and Spinal Muscular Atrophy (SMA), along with the current understanding of their genetics and mechanisms. In addition, the book details gene and cell therapies that have been developed and their translation to clinical trials. Provides an overview of gene and cell therapies for amyotrophic lateral sclerosis (ALS) and other motor neuron diseases Edited by a leading Neurosurgeon and two research scientists to promote synthesis between basic neuroscience and clinical relevance Presents a great resource for researchers and practitioners in neuroscience, neurology, and gene and cell therapy
Author: Jeffrey Laurence
Author: Jeffrey Laurence
Author: Jeffrey Laurence
Author: Kam M Hui
Author: Kelly K. Hunt
Author: National Academies of Sciences, Engineering, and Medicine
Author: 
Author: Michael G. Kaplitt
Author: Jonathan Kimmelman
Author: Odile Cohen-Haguenauer
Author: Nicholas M Boulis