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

Download or read book Fast Facts Gene Therapy written by R. Herzog and published by Karger Medical and Scientific Publishers. This book was released on 2020-04-24 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

Download or read book Gene Therapy for Human Patients written by and published by . This book was released on 1990 with total page 16 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Understanding Gene Therapy written by Prof Nick Lemoine and published by Taylor & Francis. This book was released on 2023-05-09 with total page 142 pages. Available in PDF, EPUB and Kindle. Book excerpt: This title explains what gene therapy is, how genes are delivered and how they are targeted. It discusses recent gene therapy trials, future applications and considers the ethical and safety issues surrounding gene therapy. Understanding Gene Therapy is a

Download or read book Gene Therapy written by L. E. Carmichael and published by ABDO Publishing Company. This book was released on 2013-09-01 with total page 114 pages. Available in PDF, EPUB and Kindle. Book excerpt: Amazing medical breakthroughs are made every day. In the past decades, medical researchers have cured diseases that were once deadly and devised new methods to heal that were once unimaginable. This title follows the development of gene therapy, including the discovery of DNA, groundbreaking discoveries and the doctors who made them, and where the science is heading in the future. Learn how gene therapy works and why future applications of the technology will be controversial. Sidebars, full-color photos, a glossary, and well-placed graphs, charts, and maps, enhance this engaging title. Aligned to Common Core Standards and correlated to state standards. Essential Library is an imprint of Abdo Publishing, a division of ABDO.

Download or read book Development of Gene Therapies written by Avery McIntosh and published by Chapman & Hall/CRC. This book was released on 2024 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: One of the recent advances in 21st century medicine is the emergence of gene therapies, drugs that affect the basic biology of genetic disease. The field has seen some notable setbacks in the past, but in recent years has exploded as decades of basic science have been successfully translated into the most complex biologics ever constructed, leading to regulatory approval of several gene therapy products in oncology, hematology, neurology, and ophthalmology indications. These drugs are at the apex of biological manufacturing complexity, and have the potential to be disease modifying or even curative. Evidence-based and innovative quantitative clinical development and lifecycle management strategies will be required as fixtures in the development for these unique drugs in order to reach patients in need. Development of Gene Therapies: Strategic, Scientific, and Regulatory Considerations is an unparalleled summary of the current scientific, statistical, developmental, and regulatory aspects of gene therapies, which is fast becoming a core area of the biopharmaceutical industry. This edited volume provides a systematic description of core development topics in gene therapies through 19 peer-reviewed chapters written by subject matter experts in the field. This edited volume is an invaluable resource for business leaders and investors hoping to understand the scientific principles and strategy of a company they may potentially invest in; the family members of someone affected by a genetic disease who wish to understand better how these therapies work and what they might expect as a treatment for a loved one; academic professionals, who want to learn and teach incoming medical, public health, or business students; and seasoned drug developers, who wish to learn more about the about the cutting edge of biopharmaceutical drug development. Key Features: Provides a thorough background on the scientific, manufacturing, and translational concepts and competencies for gene therapies. Covers important strategic aspects of the gene therapy industry, thereby helping investors, drug developers, and regulators gain a better appreciation of the potential value of gene therapies. Expounds on many existing and emerging state-of-the art scientific and technological advances, as well as ethical, pharmacovigilance, and regulatory considerations for gene therapy product development. Presents several case studies of successful development of gene therapies, including two of the most remarkable FDA-approved gene therapy products: Zolgensma and Luxturna. Provides perspectives and forward-looking statements on the future of gene therapies in neurological, in utero, and ultra-rare indications.

Download or read book Gene Therapy written by Theodore Friedmann and published by CSHL Press. This book was released on 1994 with total page 160 pages. Available in PDF, EPUB and Kindle. Book excerpt: In 1982, a meeting of unusual influence was held at the Banbury Conference Center of Cold Spring Harbor Laboratory. After an early attempt at treating clinical disease with transferred genes had ignited public attention and scientific controversy, a group of distinguished biologists and physicians came together to assess practical progress towards gene therapy and what its future might be. The geneticist Ted Friedmann wrote a narrative account of the participants' contributions to the meeting, ending with a personal discussion of ethical issues raised by genetic technologies. His book, the first on gene therapy, was widely read but has long been unavailable. It has been reprinted with a new introduction entitled Gene Therapy 1994, in which the author reviews the field's technical accomplishments and ethical dilemmas. Now that gene therapy has become part of the medical landscape, this volume is of interest as both a historical document and an assessment of the field's current challenges.

Download or read book Gene and Cell Therapy written by Nancy Smyth Templeton and published by CRC Press. This book was released on 2008-10-06 with total page 1154 pages. Available in PDF, EPUB and Kindle. Book excerpt: Since the publication of the second edition of this book in 2004, gene therapy and cell therapy clinical trials have yielded some remarkable successes and some disappointing failures. Now in its third edition, Gene and Cell Therapy: Therapeutic Mechanisms and Strategies assembles many of the new technical advances in gene delivery, clinical applications, and new approaches to the regulation and modification of gene expression. New Topics Covered in this Edition: Gene and Cell Therapies for Diabetes and Cardiovascular Diseases Clinical Trials Human Embryonic Stem Cells Tissue Engineering Combined with Cell Therapies Novel Polymers Relevant Nanotechnologies SiRNA Therapeutic Strategies Dendrimer Technologies Comprised of contributions from international experts, this book begins with a discussion of delivery systems and therapeutic strategies, exploring retroviral vectors and adenovirus vectors, as well as other therapeutic strategies. The middle section focuses on gene expression and detection, followed by an examination of various therapeutic strategies for individual diseases, including hematopoietic disorders, cardiovascular conditions, cancer, diabetes, cystic fibrosis, neurological disorders, and childhood-onset blindness. The final section discusses recent clinical trials and regulatory issues surrounding the new technology. This compendium is assembled by noted molecular biologist and biochemist Nancy Smyth Templeton. Baylor College of Medicine and several other institutions have used Dr. Templeton’s non-viral therapeutics in clinical trials for the treatment of lung, breast, head and neck, and pancreatic cancers, as well as Hepatitis B and C. She continues to work at the forefront of research in gene and cell therapies. Her contributions, as well as those contained in this volume, are sure to advance the state of the art of these revolutionary life-saving technologies.

Download or read book Adenoviral Vectors for Gene Therapy written by David T. Curiel and published by Academic Press. This book was released on 2016-03-10 with total page 870 pages. Available in PDF, EPUB and Kindle. Book excerpt: Adenoviral Vectors for Gene Therapy, Second Edition provides detailed, comprehensive coverage of the gene delivery vehicles that are based on the adenovirus that is emerging as an important tool in gene therapy. These exciting new therapeutic agents have great potential for the treatment of disease, making gene therapy a fast-growing field for research. This book presents topics ranging from the basic biology of adenoviruses, through the construction and purification of adenoviral vectors, cutting-edge vectorology, and the use of adenoviral vectors in preclinical animal models, with final consideration of the regulatory issues surrounding human clinical gene therapy trials. This broad scope of information provides a solid overview of the field, allowing the reader to gain a complete understanding of the development and use of adenoviral vectors. - Provides complete coverage of the basic biology of adenoviruses, as well as their construction, propagation, and purification of adenoviral vectors - Introduces common strategies for the development of adenoviral vectors, along with cutting-edge methods for their improvement - Demonstrates noninvasive imaging of adenovirus-mediated gene transfer - Discusses utility of adenoviral vectors in animal disease models - Considers Federal Drug Administration regulations for human clinical trials

Download or read book Gene Therapy written by Joseph Panno and published by Facts on File. This book was released on 2005-01 with total page 172 pages. Available in PDF, EPUB and Kindle. Book excerpt: Discusses how gene therapy works, what diseases may be treated by it, what the moral and ethical issues are, and provides case studies of Ashi DeSilva and Jesse Gelsinger.

Download or read book Gene Therapy written by Blankenstein and published by Birkhäuser. This book was released on 1999 with total page 400 pages. Available in PDF, EPUB and Kindle. Book excerpt: K. Sikora Gene therapy is one of the fastest developing areas in modern medical research. Transcending the classical preclinical and clinical disciplines, it is likely to have far reaching consequences in the practice of medicine, as we enter the next millennium. Currently, there are over 200 seperate active clinical trials with over 2,500 patients entered. These studies involve over 20 countries and include patients with a wide range of diseases, including cancer, HIV infection; cystic fibrosis (CF), haemophilia, diabetes, immune deficiencies, metabolic disorders, ischaemic heart disease and arthritis. Gene therapy can be defined as the deliberate transfer of DNA for therapeutic purposes. There is a further implication that only specific sequences containing rel evant genetic information are used; otherwise, transplantation procedures involving bone marrow, kidney or liver could be considered a form of gene therapy. The con cept of transfer of genetic information as a practical clinical tool arose from the gene-cloning technology, developed during the 1970s. Without the ability to isolate and replicate defined genetic sequences, it would be impossible to produce purified material for clinical use. The drive for the practical application of this technology came from the biotechnology industry with its quest for complex human biomole cules produced by recombinant techniques in bacteria. Within a decade, pharma ceutical-grade insulin, interferon, interleukin 2 and tumour necrosis factor were all involved in clinical trials. The next step was to obtain gene expression in vivo.

Download or read book A Guide to Human Gene Therapy written by Roland W. Herzog and published by World Scientific Publishing Company. This book was released on 2010-06 with total page 416 pages. Available in PDF, EPUB and Kindle. Book excerpt: Ever since the birth of molecular biology, the tantalizing possibility of treating disease at its genetic roots has become increasingly feasible. Gene therapy -- though still in its infancy -- remains one of the hottest areas of research in medicine. Its approach utilizes a gene transfer vehicle ("vector") to deliver therapeutic DNA or RNA to cells of the body in order to rectify the defect that is causing the disease. Successful therapies have been reported in humans in recent years such as cures in boys with severe immune deficiencies. Moreover, gene therapy strategies are being adapted in numerous biomedical laboratories to obtain novel treatments for a variety of diseases and to study basic biological aspects of disease. Correction of disease in animal studies, is steadily gaining ground, highlighting the immense potential of gene therapy in the medical profession. This book will cover topics that are at the forefront of biomedical research such as RNA interference, viral and non-viral gene transfer systems, treatment of hematological diseases and disorders of the central nervous system. Leading experts on the respective vector or disease will contribute the individual chapters and explain cutting-edge technologies. It also gives a broad overview of the most important gene transfer vectors and most extensively studied target diseases. This comprehensive guide is therefore a must-read for anyone in the biotechnology, biomedical or medical industries seeking to further their knowledge in the area of human gene therapy.

Download or read book Gene Therapy written by Clay Farris Naff and published by Greenhaven Press. This book was released on 2005 with total page 228 pages. Available in PDF, EPUB and Kindle. Book excerpt: Describes genes and gene manipulation, the history of gene therapy, the business of gene manipulation and therapy, and the ethical considerations of the procedures.

Download or read book Gene Therapy written by Ryan Kirk and published by Emereo Pty Limited. This book was released on 2014-01-20 with total page 268 pages. Available in PDF, EPUB and Kindle. Book excerpt: There has never been a Gene Therapy Guide like this. It contains 257 answers, much more than you can imagine; comprehensive answers and extensive details and references, with insights that have never before been offered in print. Get the information you need--fast! This all-embracing guide offers a thorough view of key knowledge and detailed insight. This Guide introduces what you want to know about Gene Therapy. A quick look inside of some of the subjects covered: History of medicine - Post-World War II, Neuroethics - Neuroethics of Stem Cell therapy, Gene therapy - 2009, Stem cells - Treatments, Gene therapy - Approach, Vectors in Gene Therapy - Electroporation, Medical genetics - Treatments, Biomedicine, Lipoprotein lipase deficiency - Treatment, Gene therapy for color blindness - Safety, Genetically modified virus - Gene therapy, Human genetic engineering - 1970s and earlier, Artificial pancreas - Gene therapy approach, Adeno-associated virus - Clinical trials, Genetic engineering - Medicine, Gene therapy - Deaths, Genetic disorder - Prognosis and treatment of genetic disorders, Medical genetics - Other examples, Stem cell treatments - Hematopoiesis (blood-cell formation), Gene therapy - Preventive gene therapy, Adeno-associated virus - Cell-mediated, Gene therapy - 2010, Lentivirus - Practical applications, Viral vector - Adeno-associated viruses, Alipogene tiparvovec, Human genetic engineering - Approach, Gene therapy - Vectors in gene therapy, Basal ganglia disease - Gene Therapy, Foundation Fighting Blindness - Research and Clinical Trials, Transgene - History, Stem cell controversy - Background, Neurotechnology - Future technologies, Case Western Reserve University Research, Timeline of biology and organic chemistry - 1990-present, Human genetic engineering - 2006, and much more...

Download or read book The Gene Therapy Plan written by Mitchell L. Gaynor, MD and published by Penguin Books. This book was released on 2016-04-05 with total page 370 pages. Available in PDF, EPUB and Kindle. Book excerpt: Dr. Mitchell Gaynor, integrative medicine pioneer, details what we can each do individually to keep our bodies healthy.

Download or read book Handbook of Cell and Gene Therapy written by Hazel G. Aranha and published by . This book was released on 2023 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: "This handbook provides an in-depth review of information across the developmental spectrum of gene and cell therapy products. From introductory information to state-of-the-art technologies and concepts, the book provides insights into upstream processes such as vector design and construction, purification, formulation and fill/finish as well as delivery options. Planning steps for compliance with current good manufacturing practice (CGMP) to readiness for chemistry, manufacturing, and controls (CMC) are also discussed. This book wraps up with examples of successes and pitfalls addressed by experts who have navigated the multiple challenges that are part of any innovative endeavor"--

Download or read book Fast Facts Leukemia written by Justin Loke and published by Karger Medical and Scientific Publishers. This book was released on 2022-02-22 with total page 138 pages. Available in PDF, EPUB and Kindle. Book excerpt: Leukemia is a hematologic malignancy arising from hematopoietic stem cells (HSCs) in the bone marrow. Starting with a detailed description of hematopoiesis and what goes wrong in leukemia, this concise guide covers all aspects of the four most common subtypes of the disease. Although the incidence and prevalence of leukemia are rising worldwide, survival rates are also increasing. However, both the effects of the disease and the adverse effects of treatment remain complex challenges. Yet, as our understanding of the molecular landscape increases, therapeutic options are becoming more personalized. This revised and updated second edition of 'Fast Facts: Leukemia' addresses the causes and risk factors for each subtype of leukemia, the initial and confirmatory diagnostic tests, and the latest treatment options. Designed as a comprehensive primer for physician assistants, nurse practitioners, primary care providers, oncology nurses, hematology/ oncology trainees and pharmacists, this resource will help the non-specialist and those in training to identify leukemia early and provide a thorough understanding of the pathology and genetic basis of the disease, treatment options, and effective approaches to emergency and supportive care. Table of Contents: • Understanding blood and its components • What is leukemia? • Epidemiology, etiology and risk factors • Diagnosis • Staging and general management • Supportive care • Emergencies in leukemia