Emerging Therapeutic Approaches for Cystic Fibrosis PDF Download
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Author: Miquéias Lopes-Pacheco
Publisher: Frontiers Media SA
ISBN: 2889633675
Category :
Languages : en
Pages : 281
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Book Description
Author: Miquéias Lopes-Pacheco
Publisher: Frontiers Media SA
ISBN: 2889633675
Category :
Languages : en
Pages : 281
Get Book Here
Book Description
Author: John Engelhardt
Publisher: MDPI
ISBN: 303943683X
Category : Medical
Languages : en
Pages : 208
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Book Description
Summary of Genes. Thirty years ago, the gene responsible for cystic fibrosis (CF), a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator gene, was identified. This progress has considerably changed our understanding of the pathophysiology of CF and has paved the way for the development of novel and specific therapies for the disease. The CFTR gene contains 27 exons and is characterized by a frequent three base pair deletion of the p.Phe508del. As a result of collaborative work, today more than 2000 mutations have been reported in the gene, and their impact on protein function is now more evident and useful in designing new strategies to correct the gene defect. The field of gene therapy, as illustrated by Ziying Yan in this book, has worked on identifying an efficient vector system for the delivery of the wild-type CFTR gene to the lung. At the same time, animal models have been developed in mice, rats, rabbits, zebrafish, ferrets, and pigs to establish the efficacity of gene delivery. These animals are also of the utmost importance in testing new molecules as modulators or correctors to improve the CFTR lung function. During the last three decades, the epidemiology of CF has dramatically changed, as today cystic fibrosis is now a chronic adult pulmonary disease.
Author: Venkataramana K Sidhaye
Publisher: Academic Press
ISBN: 0128038810
Category : Medical
Languages : en
Pages : 277
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Book Description
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
Author: William R. Skach
Publisher: Springer Science & Business Media
ISBN: 1592591876
Category : Medical
Languages : en
Pages : 626
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Book Description
Since the cloning of the cystic fibrosis transmembrane conductance re- lator (CFTR) nearly a decade ago, cystic fibrosis (CF) research has witnessed a dramatic expansion into new scientific areas. Basic researchers, clinicians, and patients increasingly rely on fundamental techniques of genetics, molecular biology, electrophysiology, biochemistry, cell biology, microbiology, and immunology to understand the molecular basis of this complex disease. Research into the pathophysiology of CF has established numerous paradigms of ion channel dysfunction that extend from inflammation and infection in the airways of patients to basic mechanisms of protein processing and regulation in intracellular components. With these rapid advances has come an increasing need for research scientists to understand and utilize a growing array of basic laboratory tools. This volume of Methods in Molecular Medicine, Cystic Fibrosis Methods and Protocols satisfies that need by providing detailed protocols for the laboratory techniques used throughout CF research. From electrophysiology and cell biology, to animal models and gene therapy, the comprehensive set of methods covered here provide step-by-step instructions needed for investigators to incorporate new approaches into their research programs. Contributions have been chosen to reflect the rich diversity of techniques and to provide a cohesive framework for understanding challenges that are currently at the forefront of CF research. It is hoped that this volume will serve as a valuable reference that will not only foster interdisciplinary investigations into current problems encountered in CF, but also facilitate the translation of new scientific discoveries into clinical solutions.
Author: P.L. Chang
Publisher: CRC Press
ISBN: 1351093665
Category : Science
Languages : en
Pages : 242
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Book Description
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.
Author: Kevin L. Kirk
Publisher: Springer Science & Business Media
ISBN: 9780306478376
Category : Science
Languages : en
Pages : 236
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Book Description
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.
Author: Rob Elles
Publisher: Springer Science & Business Media
ISBN: 1592594328
Category : Medical
Languages : en
Pages : 392
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Book Description
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.
Author: Prashant Mohite
Publisher: BoD – Books on Demand
ISBN: 1838810730
Category : Medical
Languages : en
Pages : 126
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Book Description
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.
Author: Stephanie Duggins Davis
Publisher: Springer Nature
ISBN: 3030423824
Category : Medical
Languages : en
Pages : 525
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Book Description
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.
Author: John R. Riordan
Publisher: Anchor Books
ISBN: 9781936113347
Category : Medical
Languages : en
Pages : 0
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Book Description
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.
