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| cystic fibrosis gene therapy: Emerging Therapeutic Approaches for Cystic Fibrosis Miquéias Lopes-Pacheco, Nicoletta Pedemonte, Anthony Kicic, 2020-01-27 |
| cystic fibrosis gene therapy: Gene Therapy Technologies, Applications and Regulations Anthony Meager, 1999 This text brings together the current data previously separated. It combines information on gene therapy technologies and the attendant regulatory requirements for gene therapy, products and procedures. |
| cystic fibrosis gene therapy: Translating Gene Therapy to the Clinic Jeffrey Laurence, Michael Franklin, 2014-11-14 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 |
| cystic fibrosis gene therapy: Cystic Fibrosis Prashant Mohite, Anna Reed, André Simon, 2021-06-09 Cystic fibrosis, a genetic disorder in children and young adults, is a multisystemic disease that mainly affects the lungs. Advances and improvements in the diagnosis and management of this condition have led to increased overall and symptom-free survival in cystic fibrosis patients. This book examines recent advances in the field and presents an evidence-based approach to the management of cystic fibrosis. |
| cystic fibrosis gene therapy: Exploring Novel Clinical Trial Designs for Gene-Based Therapies National Academies of Sciences, Engineering, and Medicine, Health and Medicine Division, Board on Health Sciences Policy, Forum on Regenerative Medicine, 2020-08-27 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. |
| cystic fibrosis gene therapy: Immunopharmacology Manzoor M. Khan, 2008-12-19 During the past decades, with the introduction of the recombinant DNA, hybridoma and transgenic technologies there has been an exponential evolution in understanding the pathogenesis, diagnosis and treatment of a large number of human diseases. The technologies are evident with the development of cytokines and monoclonal antibodies as therapeutic agents and the techniques used in gene therapy. Immunopharmacology is that area of biomedical sciences where immunology, pharmacology and pathology overlap. It concerns the pharmacological approach to the immune response in physiological as well as pathological events. This goals and objectives of this textbook are to emphasize the developments in immunology and pharmacology as they relate to the modulation of immune response. The information includes the pharmacology of cytokines, monoclonal antibodies, mechanism of action of immune-suppressive agents and their relevance in tissue transplantation, therapeutic strategies for the treatment of AIDS and the techniques employed in gene therapy. The book is intended for health care professional students and graduate students in pharmacology and immunology. |
| cystic fibrosis gene therapy: Somatic Gene Therapy P.L. Chang, 2018-01-17 As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy. |
| cystic fibrosis gene therapy: Adenoviral Vectors for Gene Therapy David T. Curiel, 2016-03-10 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 |
| cystic fibrosis gene therapy: Cystic Fibrosis in the Light of New Research Dennis Wat, 2015-08-24 Cystic Fibrosis in the Light of New Research provides the latest research and clinical evidence that will be useful for clinicians, scientists and researchers to further their knowledge around this fascinating condition. The authors have brought along their expertise and wealth of knowledge to produce this book, including the basic science that underlies the disease, the burden of bacterial and viral infections, immunologic aspects of CF, a variety of clinical measurements to predict prognosis and novel therapies including gene therapy. This book will be invaluable and entertaining for anyone who is involved in the care of patients with cystic fibrosis. |
| cystic fibrosis gene therapy: Facts about Cystic Fibrosis , 1995 |
| cystic fibrosis gene therapy: Pharmaceutical Biotechnology Daan J. A. Crommelin, Robert D. Sindelar, 2002-11-14 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. |
| cystic fibrosis gene therapy: Mammalian Artificial Chromosomes Vittorio Sgaramella, Sandro Eridani, 2008-02-02 In 1996, we organized a workshop, inter alia, at the National Research Co- cil in Milan under the generous sponsorship of the European Science Foun- tion. On that occasion, a small group of investigators convened from many countries and presented early evidence of the possibility of assembling basic units of mammalian chromosomes into artificial constructs (or, indeed, red- ing the relevant components to more manageable dimensions and defined c- stitution). Progress in the following years has been slow but steady. Many scientists who took part in the workshop have since been engaged in active and prod- tive research. It goes to the credit of Humana Press to have realized the need for a book on artificial chromosomes that aims to provide better tools to all scientists committed to this field who are confronted with very difficult tech- cal problems. We have strived to cover in Mammalian Artificial Chromosomes: Methods and Protocols all relevant areas of artificial chromosome research, from basic genetics to daring attempts to build new tools for genetic therapy. We are of course grateful to the authors who have accepted the task of describing the technical steps and pitfalls that can be encountered in their research. Rarely has a very delicate methodology been presented with such meticulous care. We have been helped in this enterprise by the excellent librarian of the LITA Institute in Segrate, Italy, Ms. Claudia Piergigli, whom we thank warmly. Ms. |
| cystic fibrosis gene therapy: Gene Therapy Protocols Paul D. Robbins, 1997 Thirty protocols by leading researchers describe in detail all the essential molecular methods for working with gene transfer systems, along with the methods for gene transfer to specific tissue types either in vivo or ex vivo. The easily reproducible methods range from those for specific viral and nonviral for both genetic and acquired diseases, to those concerned with gene delivery to particular tissues. Methods for applying specific therapeutic systems, such as ribozymes and tumor supressor genes for the treatment of AIDS and cancer, are also included in this authoritative collection. |
| cystic fibrosis gene therapy: Heritable Human Genome Editing The Royal Society, National Academy of Sciences, National Academy of Medicine, International Commission on the Clinical Use of Human Germline Genome Editing, 2021-01-16 Heritable human genome editing - making changes to the genetic material of eggs, sperm, or any cells that lead to their development, including the cells of early embryos, and establishing a pregnancy - raises not only scientific and medical considerations but also a host of ethical, moral, and societal issues. Human embryos whose genomes have been edited should not be used to create a pregnancy until it is established that precise genomic changes can be made reliably and without introducing undesired changes - criteria that have not yet been met, says Heritable Human Genome Editing. From an international commission of the U.S. National Academy of Medicine, U.S. National Academy of Sciences, and the U.K.'s Royal Society, the report considers potential benefits, harms, and uncertainties associated with genome editing technologies and defines a translational pathway from rigorous preclinical research to initial clinical uses, should a country decide to permit such uses. The report specifies stringent preclinical and clinical requirements for establishing safety and efficacy, and for undertaking long-term monitoring of outcomes. Extensive national and international dialogue is needed before any country decides whether to permit clinical use of this technology, according to the report, which identifies essential elements of national and international scientific governance and oversight. |
| cystic fibrosis gene therapy: Gene Therapy for Diseases of the Lung Kenneth Brigham, 1997-04-10 This up-to-the-minute and comprehensive resource lucidly covers gene therapy for lung diseases from existing technologies delivering foreign DNA to the lungs via the airways or circulation to promising new approaches for the further development of safe and efficient gene delivery systems. |
| cystic fibrosis gene therapy: Lung Epithelial Biology in the Pathogenesis of Pulmonary Disease Venkataramana K Sidhaye, Michael Koval, 2017-03-09 Lung Epithelial Biology in the Pathogenesis of Pulmonary Disease provides a one-stop resource capturing developments in lung epithelial biology related to basic physiology, pathophysiology, and links to human disease. The book provides access to knowledge of molecular and cellular aspects of lung homeostasis and repair, including the molecular basis of lung epithelial intercellular communication and lung epithelial channels and transporters. Also included is coverage of lung epithelial biology as it relates to fluid balance, basic ion/fluid molecular processes, and human disease. Useful to physician and clinical scientists, the contents of this book compile the important and most current findings about the role of epithelial cells in lung disease. Medical and graduate students, postdoctoral and clinical fellows, as well as clinicians interested in the mechanistic basis for lung disease will benefit from the books examination of principles of lung epithelium functions in physiological condition. - Provides a single source of information on lung epithelial junctions and transporters - Discusses of the role of the epithelium in lung homeostasis and disease - Includes capsule summaries of main conclusions as well as highlights of future directions in the field - Covers the mechanistic basis for lung disease for a range of audiences |
| cystic fibrosis gene therapy: Cystic Fibrosis Richard Spilsbury, 2018-07-15 Cystic fibrosis is a genetic metabolic disorder that affects the respiratory and digestive systems. Although the mortality rate for infants and children with cystic fibrosis used to be very high, more than half of all patients with cystic fibrosis now survive into adulthood thanks to the development of treatments such as gene therapy. This book explains the symptoms, causes, and treatments of cystic fibrosis, as well as highlighting the experiences of people who have the disease. |
| cystic fibrosis gene therapy: Adeno-Associated Virus (AAV) Vectors in Gene Therapy Kenneth I. Berns, Catherine Giraud, 2012-12-06 Human gene therapy holds great promise for the cure of many genetic diseases. In order to achieve such a cure there are two requirements. First, the affected gene must be cloned, its se quence determined and its regulation adequately characterized. Second, a suitable vector for the delivery of a good copy of the affected gene must be available. For a vector to be of use several attributes are highly desirable: these include ability to carry the intact gene (although this may be either the genomic or the cDNA form) in a stable form, ability to introduce the gene into the desired cell type, ability to express the introduced gene in an appropriately regulated manner for an extended period of time, and a lack of toxicity for the recipient. Also of concern is the frequency of cell transformation and, in some cases, the ability to introduce the gene into nondividing stem cells. Sev eral animal viruses have been tested as potential vectors, but none has proven to have all the desired properties described above. For example, retroviruses are difficult to propagate in sufficient titers, do not integrate into nondividing cells, and are of concern because of their oncogenic properties in some hosts and because they integrate at many sites in the genome and, thus, are potentially insertional mutagens. Additionally, genes introduced by retroviral vectors are frequently expressed for relatively short periods of time. A second virus used as a vector in model systems has been adenovirus (Ad). |
| cystic fibrosis gene therapy: The Cystic Fibrosis Transmembrane Conductance Regulator Kevin L. Kirk, David C. Dawson, 2003-10-31 The CFTR chloride channel is one of the most well studied transport proteins in biology. Yet there remain many mysteries about the functional properties and biological roles of this ABC transporter. The Cystic Fibrosis Transmembrane Conductance Regulator addresses a select series of `hot' topics that relate to the function of CFTR, and the links between CFTR dysfunction and human disease (i.e., cystic fibrosis). The timeliness of these topics distinguishes this collection from previous volumes of this type. Given the general interest in CFTR, this collection will appeal to a broad readership with interests in CFTR, cystic fibrosis, ion channels and ABC transporters. |
| cystic fibrosis gene therapy: Liver Disease in Children Frederick J. Suchy, Ronald J. Sokol, William F. Balistreri, 2007-05-07 Completely revised new edition of the premier reference on pediatric liver disease. Liver Disease in Children, 3rd Edition provides authoritative coverage of every aspect of liver disease affecting infants, children, and adolescents. The book offers an integrated approach to the science and clinical practice of pediatric hepatology and charts the substantial progress in understanding and treating these diseases. Chapters are written by international experts and address the unique pathophysiology, manifestations, and management of these disorders in the pediatric population. The third edition has been thoroughly updated and features new contributions on liver development, cholestatic and autoimmune disorders, fatty liver disease, and inborn errors of metabolism. With the continued evolution of pediatric hepatology as a discipline, this text remains an essential reference for all physicians involved in the care of children with liver disease. |
| cystic fibrosis gene therapy: Gene Therapy of Cancer Peter Walden, Uwe Trefzer, Wolfram Sterry, Farzin Farzaneh, 2012-12-06 The book provides a comprehensive overview of the current state, and the new concepts for the future directions of modern cancer therapy. Bringing together all the relevant aspects from basic and applied science, and the clinical experiences of this new direction in medicine, it is an up-to-date summary of the activities in the field and will be the basis for evaluating future progress in this area. |
| cystic fibrosis gene therapy: Textbook of Gene Therapy Kewal K. Jain, 1998 During the past eight years following initial gene therapy experiments, more than 200 clinical protocols have been submitted worldwide and more than 2000 patients have been treated. Although a lot remains to be accomplished before gene therapy becomes a standard medical practice, enough information has accumulated to justify a textbook on the subject. Some of the procedures and products are expected to enter the marketplace and medical practice by the year 2000. The public is very much aware of the potential of gene therapy and the medical profession should prepare itself for the new developments by learning about the basics and applications of these techniques. Much of there-search has been conducted in the bio-pharmaceutical industry by gene therapy companies and the commercial opportunities for gene therapy are enormous. A large segment of the society thus has an interest in gene therapy. This book presents a unique critical review of the tremendous progress which has been made in this field. The textbook is an effort to bring a wide range of important developments together in an accessible format. This book is aimed at physicians, gene therapists, molecular biologists, nurse practitioners and students in these fields, as well as other healthcare professionals interested in developments in the field of gene therapy and its impact, both short- and long-term, on the practice of medicine. Industrial executives planning long-term strategies in gene therapy will find this handy textbook to be a comprehensive source of information on the subject and the companies involved in developing it. Prof. K. K. Jain is a neurosurgeon trained in Canada and the United States. He has heldacademic positions in major medical centers around the world. For the last decade, he has been a consultant to a number of major biopharmaceutical companies and now runs his own biotechnology company specializing in gene therapy research and documentation. |
| cystic fibrosis gene therapy: A Guide to Human Gene Therapy Roland W. Herzog, Sergei Zolotukhin, 2010 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 |
| cystic fibrosis gene therapy: Cystic Fibrosis John R. Riordan, Richard C. Boucher, Paul M. Quinton, 2013 Cystic fibrosis is caused by mutations in the CFTR gene, which encodes a protein channel that regulates salt movement across the epithelial membranes of the lungs, pancreas, and other organs. In cystic fibrosis patients, salt transport is impeded, causing sticky, viscous mucus to build up and clog these vital organs. Written and edited by experts in the field, this collection from Cold Spring Harbor Perspectives in Medicine provides an in-depth examination of cystic fibrosis biology and treatment strategies. Contributors examine the structure and dynamics of CFTR, its normal physiological roles in the airway and digestive epithelia, and how those operations are impaired in patients with cystic fibrosis. The numerous CFTR mutations and how they alter the expression, synthesis, processing, and function of CFTR in cystic fibrosis and other CFTR-related disorders are considered, as are disease- modifying genes that influence disease severity. This volume includes discussions of therapy and treatment strategies for cystic fibrosis, such as airway clearance techniques, CFTR modulation or supplementation, and pancreatic enzyme replacements. It will be an essential reference for molecular and cellular biologists, physiologists, and clinicians interested in understanding the biological basis of the disease and the search for effective therapies. |
| cystic fibrosis gene therapy: Cystic Fibrosis Stephanie Duggins Davis, Margaret Rosenfeld, James Chmiel, 2020-05-21 This book provides a comprehensive overview of the multisystem disease, cystic fibrosis, for both pediatric and adult patients. Written by experts in the field, the text outlines the progressive nature of CF as well as the impact of this autosomal recessive disease on the respiratory, gastrointestinal, endocrine, rheumatologic, and renal systems, as well as the patient’s mental health. The book begins with a chapter describing the history of cystic fibrosis and how the face of this life-shortening disease has changed over the past several decades. The following chapters elucidate the pathophysiology of how cystic fibrosis impacts each organ system. Current management and therapeutics are detailed with step-by-step guidelines for clinicians. This book is unique in that it highlights the entire person, not just the respiratory system, with detailed inclusion of the patient perspectives throughout, informing practice standards and considerations. This is an ideal guide for pediatric and adult physicians who care for patients with cystic fibrosis, as well as respiratory therapists, physical therapists, nurses, nutritionists, and pharmacists who care for these patients. |
| cystic fibrosis gene therapy: Introduction to Basics of Pharmacology and Toxicology Gerard Marshall Raj, Ramasamy Raveendran, 2019-11-16 This book illustrates, in a comprehensive manner, the most crucial principles involved in pharmacology and allied sciences. The title begins by discussing the historical aspects of drug discovery, with up to date knowledge on Nobel Laureates in pharmacology and their significant discoveries. It then examines the general pharmacological principles - pharmacokinetics and pharmacodynamics, with in-depth information on drug transporters and interactions. In the remaining chapters, the book covers a definitive collection of topics containing essential information on the basic principles of pharmacology and how they are employed for the treatment of diseases. Readers will learn about special topics in pharmacology that are hard to find elsewhere, including issues related to environmental toxicology and the latest information on drug poisoning and treatment, analytical toxicology, toxicovigilance, and the use of molecular biology techniques in pharmacology. The book offers a valuable resource for researchers in the fields of pharmacology and toxicology, as well as students pursuing a degree in or with an interest in pharmacology. |
| cystic fibrosis gene therapy: Breath from Salt Bijal P. Trivedi, 2020-09-08 Recommended by Bill Gates and included in GatesNotes Elaborating on the science as well as the business behind the fight against cystic fibrosis, Trivedi captures the emotions of the families, doctors, and scientists involved in the clinical trials and their 'weeping with joy' as new drugs are approved, and shows how cystic fibrosis, once a 'death sentence,' became, for many, a manageable condition. This is a rewarding and challenging work. —Publishers Weekly Cystic fibrosis was once a mysterious disease that killed infants and children. Now it could be the key to healing millions with genetic diseases of every type—from Alzheimer's and Parkinson's to diabetes and sickle cell anemia. In 1974, Joey O'Donnell was born with strange symptoms. His insatiable appetite, incessant vomiting, and a relentless cough—which shook his tiny, fragile body and made it difficult to draw breath—confounded doctors and caused his parents agonizing, sleepless nights. After six sickly months, his salty skin provided the critical clue: he was one of thousands of Americans with cystic fibrosis, an inherited lung disorder that would most likely kill him before his first birthday. The gene and mutation responsible for CF were found in 1989—discoveries that promised to lead to a cure for kids like Joey. But treatments unexpectedly failed and CF was deemed incurable. It was only after the Cystic Fibrosis Foundation, a grassroots organization founded by parents, formed an unprecedented partnership with a fledgling biotech company that transformative leaps in drug development were harnessed to produce groundbreaking new treatments: pills that could fix the crippled protein at the root of this deadly disease. From science writer Bijal P. Trivedi, Breath from Salt chronicles the riveting saga of cystic fibrosis, from its ancient origins to its identification in the dank autopsy room of a hospital basement, and from the CF gene's celebrated status as one of the first human disease genes ever discovered to the groundbreaking targeted genetic therapies that now promise to cure it. Told from the perspectives of the patients, families, physicians, scientists, and philanthropists fighting on the front lines, Breath from Salt is a remarkable story of unlikely scientific and medical firsts, of setbacks and successes, and of people who refused to give up hope—and a fascinating peek into the future of genetics and medicine. |
| cystic fibrosis gene therapy: Gene Therapy Methods M. Ian Phillips, 2002-02-22 This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series. Discusses methods currently used in preclinical and clinical gene therapy Covers the use of adeno-associated virus delivery for treatment of Parkinson's disease |
| cystic fibrosis gene therapy: Advances in Biotechnology Indu Ravi, Mamta Baunthiyal, Jyoti Saxena, 2013-10-21 The book “Advances in Biotechnology” is about recent advances in some of the important fields that are ongoing in certain biotechnological applications. Biotechnology has been quite helpful in keeping pace with the demands of every increasing human population and in improving the quality of human life. Major biotechnological achievements associated with human welfare have been from the fields like genetic engineering; transgenic plants and animals; genomics, proteomics, monoclonal antibodies for the diagnosis of disease, gene therapy etc. Fourteen authoritative chapters written by experts having experience in academics and research on current developments and future trends in biotechnology have been empathized. The book provides a detailed account of various methodologies used in biotechnology i.e. High capacity vectors, DNA sequencing dealing with next generation sequencing, Molecular markers, DNA microarray technology, as well as Proteomics that have revolutionized biotechnology with a wide array of applications. The book not only presents a well-founded explanation of the topics but also aims to present up-to-date reviews of current research efforts, some thoughtful discussions on the potential benefits and risks involved in producing biotechnological products and the challenges of bringing such products to market. It will prove to be an excellent reference work for both academicians and researchers, indicating new starting points to young researchers for new projects in the field. The book is intended for biotechnologist, biologist, researchers, teachers and students of Biosciences and Biotechnology. |
| cystic fibrosis gene therapy: Gene Therapy of Autoimmune Disease Gerald J. Prud'homme, 2005-07-13 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. |
| cystic fibrosis gene therapy: A Life Course Perspective on Health Trajectories and Transitions Claudine Burton-Jeangros, Stéphane Cullati, Amanda Sacker, David Blane, 2015-08-11 This open access book examines health trajectories and health transitions at different stages of the life course, including childhood, adulthood and later life. It provides findings that assess the role of biological and social transitions on health status over time. The essays examine a wide range of health issues, including the consequences of military service on body mass index, childhood obesity and cardiovascular health, socio-economic inequalities in preventive health care use, depression and anxiety during the child rearing period, health trajectories and transitions in people with cystic fibrosis and oral health over the life course. The book addresses theoretical, empirical and methodological issues as well as examines different national contexts, which help to identify factors of vulnerability and potential resources that support resilience available for specific groups and/or populations. Health reflects the ability of individuals to adapt to their social environment. This book analyzes health as a dynamic experience. It examines how different aspects of individual health unfold over time as a result of aging but also in relation to changing socioeconomic conditions. It also offers readers potential insights into public policies that affect the health status of a population. |
| cystic fibrosis gene therapy: Cystic Fibrosis Alex Horsley, Steve Cunningham, J. Alastair Innes, 2015 Cystic Fibrosis (CF) is a multi-system disorder, requiring not just respiratory expertise but also management of nutrition, and diabetes, as well psychosocial issues. This pocketbook will be a concise companion for all health care professionals in respiratory medicine, paediatrics, and primary care who manage, or come across, patients with CF. The book will cover all aspects of care, including both paediatric and adult-specific issues and summarize up-to-date literature in a concise and focussed style. There will be an emphasis on the practical aspects of management with the effects of CF in the lung, the microbiology of pulmonary CF, and management of exacerbations covered in separate chapters. The psychosocial aspects of CF care, end of life care and lung transplantation will also be addressed, and potential future therapies reviewed. This second edition will be updated to reflect the UK CF Trust Standards of Care; include emerging organisms, eg Pandorea, and treatment guidelines and Cochrane reviews; an expanded section on physiotherapy; and a new chapter on pharmacopeia. |
| cystic fibrosis gene therapy: Genetic Twists of Fate Stanley Fields, Mark Johnston, 2010-09-24 How tiny variations in our personal DNA can determine how we look, how we behave, how we get sick, and how we get well. News stories report almost daily on the remarkable progress scientists are making in unraveling the genetic basis of disease and behavior. Meanwhile, new technologies are rapidly reducing the cost of reading someone's personal DNA (all six billion letters of it). Within the next ten years, hospitals may present parents with their newborn's complete DNA code along with her footprints and APGAR score. In Genetic Twists of Fate, distinguished geneticists Stanley Fields and Mark Johnston help us make sense of the genetic revolution that is upon us. Fields and Johnston tell real life stories that hinge on the inheritance of one tiny change rather than another in an individual's DNA: a mother wrongly accused of poisoning her young son when the true killer was a genetic disorder; the screen siren who could no longer remember her lines because of Alzheimer's disease; and the president who was treated with rat poison to prevent another heart attack. In an engaging and accessible style, Fields and Johnston explain what our personal DNA code is, how a few differences in its long list of DNA letters makes each of us unique, and how that code influences our appearance, our behavior, and our risk for such common diseases as diabetes or cancer. |
| cystic fibrosis gene therapy: Cystic Fibrosis Maxine Rosaler, 2006-08-15 Describes the history and causes of cystic fibrosis, and discusses the special needs and complications that can arise. |
| cystic fibrosis gene therapy: Foundations of Respiratory Medicine Simon Hart, Mike Greenstone, 2018-09-04 This book meets the need for a resource that covers the core knowledge required to pass the SCE exam, which includes the broad field of respiratory medicine. This book is also highly applicable to core medical trainees sitting their MRCP examinations. The format is ideal for effective exam revision with individual chapters covering the key points of each condition in sufficient (but not excessive) detail. Examples of imaging (CXR, CT, PET-CT) are utilised to illustrate cases and descriptions of modern respiratory intervention such as the EBUS/EUS-guided sampling and medical thoracoscopy is included in this essential exam resource. |
| cystic fibrosis gene therapy: Molecular Diagnosis of Genetic Diseases Rob Elles, 2008-02-01 This completely revised and updated second edition to integrates the many new technologies and insights now available for the diagnosis of genetic diseases. The authors use such methodologies as PCR optimization dosage analysis, mutation scanning, and quantitative fluorescent PCR for aneuploidy analysis, Neurofibromatosis type 1, and Duchenne muscular dystrophy. These largely generic methodologies may be adapted to most genetic conditions for which a molecular diagnosis is relevant, no matter how frequent or rare their incidence. Molecular Diagnosis of Genetic Diseases, Second Edition offers diagnostic molecular geneticists a unique opportunity to sharpen their scientific skills in the design of assays, their execution, and their interpretation. |
| cystic fibrosis gene therapy: Assisted Reproduction Techniques Khaldoun Sharif, Arri Coomarasamy, 2012-03-26 Assisted reproduction techniques have led to the birth of 4 million babies worldwide Assisted reproduction techniques (ART), in particular in-vitro fertilization and intra-cytoplasmic sperm injection, are the most advanced forms of infertility treatment. They involve numerous counseling, medical, surgical and laboratory-based steps. At each step various problems and complications could be encountered that challenge even the most experienced ART practitioners. Moreover, patients with complex medical disorders may require ART, presenting further challenges. Assisted Reproduction Techniques will stimulate resourceful thinking in the ART practitioner when faced with these challenges. It outlines various management options, the reasoning behind them, and the evidence on which they are based to enable the practitioner to choose the most suitable solution for the needs of each patient. Written by 122 internationally renowned experts, Assisted Reproduction Techniques follows the patient's journey throughout the whole ART process, with chapters on: Counseling and preparation Ovarian stimulation Oocyte retrieval Embryo transfer The luteal phase The ART laboratory The male patient The ART pregnancy Each of the 100 concise chapters includes clinical cases, background, evidence-based practical management options, preventive measures and key-point summaries of the important details. Assisted Reproduction Techniques gives a wide-ranging practical guide to all those wishing to support couples who cannot conceive naturally. |
| cystic fibrosis gene therapy: Alex Frank Deford, 2015-02-24 A father’s moving memoir of cystic fibrosis “captures a brave child’s legacy as well as the continuing fight against the genetic disease” (The New York Times). In 1971 a girl named Alex was born with cystic fibrosis, a degenerative genetic lung disease. Although health-care innovations have improved the life span of CF patients tremendously over the last four decades, the illness remains fatal. Given only two years to live by her doctors, the imaginative, excitable, and curious little girl battled through painful and frustrating physical-therapy sessions twice daily, as well as regular hospitalizations, bringing joy to the lives of everyone she touched. Despite her setbacks, brave Alex was determined to live life like a typical girl—going to school, playing with her friends, traveling with her family. Ultimately, however, she succumbed to the disease in 1980 at the age of eight. Award-winning author Frank Deford, celebrated primarily as a sportswriter, was also a budding novelist and biographer at the time of his daughter’s birth. Deford kept a journal of Alex’s courageous stand against the disease, documenting his family’s struggle to cope with and celebrate the daily fight she faced. This book is the result of that journal. Alex relives the events of those eight years: moments as heartwarming as when Alex recorded herself saying “I love you” so her brother could listen to her whenever he wanted, and as heartrending as the young girl’s tragic, dawning realization of her own very tenuous mortality, and her parents’ difficulty in trying to explain why. Though Alex is a sad story, it is also one of hope; her greatest wish was that someday a cure would be found. Deford has written a phenomenal memoir about an extraordinary little girl. |
| cystic fibrosis gene therapy: Hodson and Geddes' Cystic Fibrosis Andrew Bush, Diana Bilton, Margaret Hodson, 2015-07-24 Hodson and Geddes' Cystic Fibrosis provides everything the respiratory clinician, pulmonologist or health professional treating patients needs in a single manageable volume. This international and authoritative work brings together current knowledge and has become established in previous editions as a leading reference in the field. This fourth edition includes a wealth of new information, figures, useful videos, and a companion eBook. The basic science that underlies the disease and its progression is outlined in detail and put into a clinical context. Diagnostic and clinical aspects are covered in depth, as well as promising advances such as gene therapies and other novel molecular based treatments. Patient monitoring and the importance of multidisciplinary care are also emphasized. This edition: Features accessible sections reflecting the multidisciplinary nature of the cystic fibrosis care team Contains a chapter written by patients and families about their experiences with the disease Includes expanded coverage of clinical areas, including chapters covering sleep, lung mechanics and the work of breathing, upper airway disease, insulin deficiency and diabetes, bone disease, and sexual and reproductive issues Discusses management both in the hospital and at home Includes a new section on monitoring and discusses the use of databases to improve patient care Covers monitoring in different age groups, exercise testing and the outcomes of clinical trials in these areas Includes chapters devoted to nursing, physiotherapy, psychology, and palliative and spiritual care Throughout, the emphasis is on providing an up-to-date and balanced review of both the clinical and basic science aspects of the subject and reflecting the multidisciplinary nature of the cystic fibrosis care team. |
| cystic fibrosis gene therapy: The Metabolic & Molecular Bases of Inherited Disease Charles R. Scriver, 2001 Presents clinical, biochemical, and genetic information concerning those metabolic anomalies grouped under inborn errors of metabolism. |
Cyst: Pictures, Causes, Types, Treatments, and Prevention
Feb 5, 2024 · Cystic acne can look like large, pus-filled boils on the skin. It can also be painful to the touch. If these boils rupture, they can cause scarring.
Tumor vs. cyst: What's the difference? - Mayo Clinic
Jul 22, 2023 · What's the difference between a tumor and a cyst? Could a cyst be cancerous? Tumors and cysts are two distinct entities. Cyst. A cyst is a sac that may be filled with air, fluid …
8 Types of Cysts: Causes, Symptoms & Treatment - eMedicineHealth
Some causes of cysts include impact injuries, blocked ducts, cellular defects, and parasites. A cyst is a pocket of fluid that can form in different areas of the body such as the skin, internal …
Cysts: Types, pictures, symptoms, causes, and treatment
Dec 22, 2023 · There are various different types of cysts. Cystic acne, or nodulocystic acne, is a severe type of acne in which the skin’s pores become blocked, leading to infection and …
Cyst - Wikipedia
Some cysts are neoplastic, and thus are called cystic tumors. Many types of cysts are not neoplastic, they are dysplastic or metaplastic. Pseudocysts are similar to cysts in that they …
What Is a Cyst? Types, Symptoms, Signs, and Causes - MedicineNet
A cyst is a closed sac-like structure that is not a normal part of the tissue where it is located. Cysts are common and can occur anywhere in the body in people of any age. Sometimes they may …
What is A Cystic Lesion? | Medical Insights Unveiled
Cystic lesions are abnormal growths that contain fluid or semi-solid material. They can develop in nearly any organ, including the skin, ovaries, kidneys, and liver. The fluid within these lesions …
CYSTIC Definition & Meaning - Merriam-Webster
The meaning of CYSTIC is of or relating to the urinary bladder or the gallbladder. How to use cystic in a sentence.
Cystic | definition of cystic by Medical dictionary
1. pertaining to or containing cysts. 2. pertaining to the urinary bladder or to the gallbladder. cystic disease of breast fibrocystic disease of breast.
Cystic Cysts | Causes, Signs, Symptoms, Diagnoses, Treatments
Sep 10, 2024 · Understand the Different Types of Cystic Cysts. Learn What Causes Them and When to Seek Treatment to Manage Pain and Prevent Complications.
Cyst: Pictures, Causes, Types, Treatments, and Prevention
Feb 5, 2024 · Cystic acne can look like large, pus-filled boils on the skin. It can also be painful to the touch. If these boils rupture, they can cause scarring.
Tumor vs. cyst: What's the difference? - Mayo Clinic
Jul 22, 2023 · What's the difference between a tumor and a cyst? Could a cyst be cancerous? Tumors and cysts are two distinct entities. Cyst. A cyst is a sac that may be filled with air, fluid or …
8 Types of Cysts: Causes, Symptoms & Treatment - eMedicineHealth
Some causes of cysts include impact injuries, blocked ducts, cellular defects, and parasites. A cyst is a pocket of fluid that can form in different areas of the body such as the skin, internal organs, …
Cysts: Types, pictures, symptoms, causes, and treatment
Dec 22, 2023 · There are various different types of cysts. Cystic acne, or nodulocystic acne, is a severe type of acne in which the skin’s pores become blocked, leading to infection and …
Cyst - Wikipedia
Some cysts are neoplastic, and thus are called cystic tumors. Many types of cysts are not neoplastic, they are dysplastic or metaplastic. Pseudocysts are similar to cysts in that they have …
What Is a Cyst? Types, Symptoms, Signs, and Causes - MedicineNet
A cyst is a closed sac-like structure that is not a normal part of the tissue where it is located. Cysts are common and can occur anywhere in the body in people of any age. Sometimes they may be …
What is A Cystic Lesion? | Medical Insights Unveiled
Cystic lesions are abnormal growths that contain fluid or semi-solid material. They can develop in nearly any organ, including the skin, ovaries, kidneys, and liver. The fluid within these lesions can …
CYSTIC Definition & Meaning - Merriam-Webster
The meaning of CYSTIC is of or relating to the urinary bladder or the gallbladder. How to use cystic in a sentence.
Cystic | definition of cystic by Medical dictionary
1. pertaining to or containing cysts. 2. pertaining to the urinary bladder or to the gallbladder. cystic disease of breast fibrocystic disease of breast.
Cystic Cysts | Causes, Signs, Symptoms, Diagnoses, Treatments
Sep 10, 2024 · Understand the Different Types of Cystic Cysts. Learn What Causes Them and When to Seek Treatment to Manage Pain and Prevent Complications.
