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Translating Gene Therapy To The Clinic
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Book Synopsis Translating Gene Therapy to the Clinic by : Jeffrey Laurence
Download or read book Translating Gene Therapy to the Clinic written by Jeffrey Laurence and published by Academic Press. This book was released on 2014-11-14 with total page 346 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Book Synopsis Translating Regenerative Medicine to the Clinic by : Jeffrey Laurence
Download or read book Translating Regenerative Medicine to the Clinic written by Jeffrey Laurence and published by Academic Press. This book was released on 2015-11-18 with total page 356 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Book Synopsis Gene Therapy - From Laboratory To The Clinic by : Kam M Hui
Download or read book Gene Therapy - From Laboratory To The Clinic written by Kam M Hui and published by World Scientific. This book was released on 1994-11-29 with total page 289 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Book Synopsis Nonviral Vectors for Gene Therapy by :
Download or read book Nonviral Vectors for Gene Therapy written by and published by Academic Press. This book was released on 2015-01-23 with total page 296 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Book Synopsis Gene Therapy for Cancer by : Kelly K. Hunt
Download or read book Gene Therapy for Cancer written by Kelly K. Hunt and published by Springer Science & Business Media. This book was released on 2007-10-26 with total page 469 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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 13 :0309495849 Total Pages :93 pages Book Rating :4.3/5 (94 download)
Book Synopsis Advancing Gene-Targeted Therapies for Central Nervous System Disorders by : National Academies of Sciences, Engineering, and Medicine
Download or read book Advancing Gene-Targeted Therapies for Central Nervous System Disorders written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2019-12-01 with total page 93 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Book Synopsis Gene Therapy of the Central Nervous System: From Bench to Bedside by : Michael G. Kaplitt
Download or read book Gene Therapy of the Central Nervous System: From Bench to Bedside written by Michael G. Kaplitt and published by Elsevier. This book was released on 2005-11-11 with total page 370 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
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.
Book Synopsis Fast Facts: Gene Therapy by : Roland Herzog
Download or read book Fast Facts: Gene Therapy written by Roland Herzog and published by Karger Medical and Scientific Publishers. This book was released on 2020-04-08 with total page 102 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: • Principles of gene therapy • Gene therapy techniques • Ethical and safety considerations • Gene therapies with proven clinical efficacy • Genome editing • Research directions – the next wave of treatments
Book Synopsis The Clinibook by : Odile Cohen-Haguenauer
Download or read book The Clinibook written by Odile Cohen-Haguenauer and published by EDK Editions. This book was released on 2013-05-03T00:00:00+02:00 with total page 589 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Book Synopsis Translating MicroRNAs to the Clinic by : Jeffrey Laurence
Download or read book Translating MicroRNAs to the Clinic written by Jeffrey Laurence and published by Academic Press. This book was released on 2016-09-17 with total page 392 pages. Available in PDF, EPUB and Kindle. Book excerpt: Translating microRNA to the Clinic reviews the possibilities of current methodological tools and experimental approaches used by leading translational researchers. The book features the uses of micro ribonucleic acid as deployed in cancer targeting in biomarkers, diabetes, cardiovascular disease, and neurodegeneration, among many others. Pedagogically, the work concentrates on the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in this field, promoting a cross-disciplinary communication between the sub-specialities of medicine, but in common with other books on the topic. In addition, the book emphasizes 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. Encompasses the latest innovations and tools being used to apply microRNA from lab to clinic“/li> Extensive pedagogical updates aiming to improve the education of translational researchers in this field Transdisciplinary approach aims to support cross-fertilization between different sub-specialties of medicine
Book Synopsis Pharmaceutical Biotechnology by : Daan J. A. Crommelin
Download or read book Pharmaceutical Biotechnology written by Daan J. A. Crommelin and published by CRC Press. This book was released on 2002-11-14 with total page 456 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.
Book Synopsis Molecular and Cellular Therapies for Motor Neuron Diseases by : Nicholas M Boulis
Download or read book Molecular and Cellular Therapies for Motor Neuron Diseases written by Nicholas M Boulis and published by Academic Press. This book was released on 2017-01-18 with total page 336 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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
Book Synopsis Gene Therapy, An Issue of Hematology/Oncology Clinics of North America, E-Book by : Daniel E. Bauer
Download or read book Gene Therapy, An Issue of Hematology/Oncology Clinics of North America, E-Book written by Daniel E. Bauer and published by Elsevier Health Sciences. This book was released on 2017-09-27 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: This issue of Hematology/Oncology Clinics will focus on Gene Therapy. Topics include, but are not limited to Historical Perspective and Current Renaissance, Integrating Vectors, Nonintegrating Vectors, Gene Editing, Conditioning Therapies for Autologous HSCT, Approaches to Immunodeficiency, Approaches to Hemoglobinopathy, Approaches to Hemophilia, Hematopoietic Gene Therapies for Neurologic and Metabolic Disease, Gene Therapy Approaches to HIV and other Infectious Diseases, HSC Approaches to Cancer, and Gene Modified T Cell Therapies for Cancer.
Book Synopsis A Guide to Human Gene Therapy by : Roland W. Herzog
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
Book Synopsis Gene Therapy of Cancer by : Wolfgang Walther
Download or read book Gene Therapy of Cancer written by Wolfgang Walther and published by Springer Science & Business Media. This book was released on 2008-02-01 with total page 633 pages. Available in PDF, EPUB and Kindle. Book excerpt: Since the discovery of the molecular structure of genes and the unveiling of the molecular basis of numerous human diseases, scientists have been fas- nated with the possibility of treating certain diseases by transducing foreign DNA into the affected cells. Initially, it was proposed that the foreign DNA could either replace defective nonfunctional genes, or code for therapeutic proteins. This concept has evolved into the rapidly growing field of gene therapy. Even though surgery, radiotherapy, and chemotherapy are widely ava- able and routinely used for cancer treatment, these therapies fail to cure approximately 50 percent of cancer patients. Therefore, since it is a disease characterized by aberrant gene expression, cancer has been a target of gene therapy research since the inception of this treatment modality. Numerous cancer gene therapy strategies are currently being investigated, including gene replacement therapy, the regulation of gene expression to modulate immu- logical responses to tumors, the direct killing of tumor cells, and direct int- ference with tumor growth. In this context, gene transfer systems, tumor-specific expression vectors, and novel therapeutic genes have been extensively st- ied. All these strategies aim for the selective destruction of human malignant disease while circumventing the destruction of nonmalignant cells and tissues thereby minimizing toxicity to the patient.
Book Synopsis Gene Therapy Technologies, Applications and Regulations by : Anthony Meager
Download or read book Gene Therapy Technologies, Applications and Regulations written by Anthony Meager and published by John Wiley & Sons. This book was released on 1999-11-05 with total page 448 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene Therapy Technologies, Applications and Regulations From Laboratory to Clinic Edited by Anthony Meager Division of Immunobiology, The National Institute for Biological Standards and Control, South Mimms, UK The development of gene-based technologies has been rapid over the past decade and has consequently resulted in a surge of interest in human gene therapy, the deliberate transfer of genes to somatic cells to cure or alleviate disease symptoms. Hundreds of clinical protocols involving variously designed vectors for efficient gene transfer have been developed. However, the use of such complex 'gene medicines' containing potentially heritable genes has raised numerous concerns regarding quality, efficacy and safety. Encompassing recent developments in the field and addressing current concerns this book: surveys many of the current technologies for preparing vectors for use in gene therapy protocols reviews the application of gene-mediated therapies to a range of medical conditions considers the regulatory aspects of gene therapy including product quality and safety requirements appraises the transfer of technologies from laboratory to clinic with regard to the attendant requirements and facilities for: good laboratory practice (GLP) conditions in the R&D laboratory large-scale production methods and good manufacturing practice (GMP) current in-process and final product testing Written by international experts knowledgeable about many aspects of human somatic gene therapy, this book will be an essential guide for those embarking on gene therapy technologies relevant to specifications of production and testing of products (and procedures) required to meet existing regulations, including quality, efficacy and safety considerations.
