Download or read book Emerging Therapeutic Approaches for Cystic Fibrosis written by Miquéias Lopes-Pacheco and published by Frontiers Media SA. This book was released on 2020-01-27 with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Cystic Fibrosis A Comprehensive Exploration of Pathways to Understanding Treatment and Holistic Care written by Dr. Spineanu Eugenia and published by Dr. Spineanu Eugenia. This book was released on 2024-09-21 with total page 117 pages. Available in PDF, EPUB and Kindle. Book excerpt: Dive into a comprehensive guide, 'Cystic Fibrosis: A Comprehensive Exploration of Pathways to Understanding, Treatment, and Holistic Care,' delving deep into the intricate realms of this genetic condition. Explore the genetic underpinnings, anatomical complexities, and the impact of Cystic Fibrosis on various bodily systems. Uncover insights into the multifaceted manifestations of CF, from respiratory and digestive challenges to broader implications on mental health and overall well-being. This treatise intricately dissects the genetic mutations, their biochemical implications, and the resulting dysfunction within the CFTR protein, offering a clear understanding of how this condition affects ion transport and fluid balance across organs. From the intricacies of CF's impact on the respiratory and digestive systems to its ramifications on mental health, this guide provides an in-depth exploration of symptoms, complications, and holistic management approaches.

Download or read book Cystic Fibrosis Solutions Book written by Lizzy Scott and published by Independently Published. This book was released on 2023-11-14 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Are you seeking a comprehensive guide to navigate the intricate landscape of Cystic Fibrosis care, filled with groundbreaking insights and transformative strategies? Look no further than "Precision Medicine and Personalized Care: Empowering Lives through Innovative Strategies and Cutting-Edge Therapies." This visionary book is a beacon of hope for individuals and families impacted by Cystic Fibrosis, offering a roadmap to a future where precision medicine revolutionizes treatment approaches. In the first chapters, delve into the core of Cystic Fibrosis with "Understanding Cystic Fibrosis," exploring its genetic foundations, clinical manifestations, and the profound impact on quality of life. Gain a deep understanding of the historical evolution of treatments, from traditional approaches to the pressing need for precision medicine, setting the stage for the transformative journey ahead. Embark on a journey through the heart of precision medicine in "Precision Medicine and Genetic Therapies." Discover the genomic insights unlocking the secrets of Cystic Fibrosis, paving the way for individualized treatment approaches that transcend the limitations of one-size-fits-all interventions. Uncover the potential of CRISPR-Cas9 and gene editing technologies, promising to rewrite the genetic narrative of Cystic Fibrosis and bring about groundbreaking therapeutic solutions. Chapter after chapter, witness the unveiling of "Personalized Care Strategies" that tailor treatment plans to individual needs. Explore patient-centered approaches, the integration of mental health support, and the meticulous addressing of co-morbidities, creating a holistic framework that empowers individuals with Cystic Fibrosis to live fuller, healthier lives. As you turn the pages to Chapter 5, "Cutting-Edge Therapies and Future Directions," brace yourself for a glimpse into the future of Cystic Fibrosis care. Uncover the latest innovations, from emerging therapies and the profound impact of CFTR modulators to the transformative potential of stem cell and gene therapies. Immerse yourself in the world of promising research and clinical trials, guided by the beacon of patient advocacy that shapes the trajectory of Cystic Fibrosis solutions. This book is not just a guide; it's a lifeline for those seeking empowerment and knowledge in the face of Cystic Fibrosis. Whether you are a patient, caregiver, or healthcare professional, "Precision Medicine and Personalized Care" is a must-read, offering a compelling narrative of hope, progress, and the relentless pursuit of a future where Cystic Fibrosis is not just managed but conquered. Embrace this indispensable resource and join the journey towards empowering lives through innovative strategies and cutting-edge therapies. Your path to understanding, resilience, and a brighter future for Cystic Fibrosis starts here.

Download or read book Molecular Basis and Gene Therapies of Cystic Fibrosis written by John Engelhardt and published by MDPI. This book was released on 2020-12-11 with total page 208 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Cystic Fibrosis in the Light of New Research written by Dennis Wat and published by BoD – Books on Demand. This book was released on 2015-08-24 with total page 394 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Cystic Fibrosis written by Prashant Mohite and published by BoD – Books on Demand. This book was released on 2021-06-09 with total page 126 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Molecular Basis and Gene Therapies of Cystic Fibrosis written by John Engelhardt and published by . This book was released on 2020 with total page 210 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Cystic Fibrosis in the 21st Century written by Andrew Bush and published by Karger Medical and Scientific Publishers. This book was released on 2006 with total page 341 pages. Available in PDF, EPUB and Kindle. Book excerpt: Cystic fibrosis used to be thought of as a respiratory and digestive disease, with a uniformly and rapidly fatal outcome. The spectrum of the disease has broadened into the mild atypical case, presenting in middle age, with the potential for complications in virtually every system of the body. In the past few years there has been an explosion of knowledge of the basic science of the defect. The editors have therefore invited the leading scientists and clinicians in the field of cystic fibrosis to describe the recent advances in this disease. Although there are many 'Recent Advances' texts, previous books have been selective in their choice of topics. This book is the first to cover the entire field of this complex disease, and encompasses the rapidly moving topics of the basic molecular and cellular biology as well as the recent multi-system, multi-disciplinary advances in the clinical care of patients. The authors have been charged with writing only about new developments and not to rehash old literature. The bulk of the references is therefore less than five years old. This book addresses all professionals who treat cystic fibrosis and want to have an up-date of new findings in the field, particularly of those outside their immediate specialisation. It will also be useful for basic researchers interested in related scientific areas and the clinical context of their work.

Download or read book Psychosocial Aspects of Cystic Fibrosis written by Bryan Lask and published by CRC Press. This book was released on 2001-02-02 with total page 504 pages. Available in PDF, EPUB and Kindle. Book excerpt: The treatment and care of those with cystic fibrosis has developed rapidly over the last decade, with a resulting increase in the number of individuals living well into adolescence and early adulthood. This book deals with the profound psychological impact of these changes on sufferers, their friends and families, and those involved in patient care. It also considers new approaches to problems that continue to be of concern to clinicians and other health professionals working in the field, including adherence to therapy, communication within families, staff burnout, education and counselling. The six sections include an introduction to the aetiology, pathology, course and treatment of cystic fibrosis, a series of personal perspectives, developmental considerations, the illness network, therapeutic approaches, and major contemporary issues such as transplantation, genetic screening and new therapies. Where relevant, chapters include an update of current knowledge, discussions of the clinical implications of current an future research, and guidelines for clinical practice.

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 with total page 450 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

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 Hodson and Geddes Cystic Fibrosis written by Andrew Bush and published by CRC Press. This book was released on 2015-07-24 with total page 675 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Exploring Novel Clinical Trial Designs for Gene Based Therapies written by National Academies of Sciences, Engineering, and Medicine and published by National Academies Press. This book was released on 2020-08-27 with total page 127 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Cystic Fibrosis Methods and Protocols written by William R. Skach and published by Springer Science & Business Media. This book was released on 2008-02-02 with total page 626 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Advanced Textbook On Gene Transfer Gene Therapy And Genetic Pharmacology Principles Delivery And Pharmacological And Biomedical Applications Of Nucleotide based Therapies written by Daniel Scherman and published by World Scientific. This book was released on 2013-12-10 with total page 557 pages. Available in PDF, EPUB and Kindle. Book excerpt: This unique advanced textbook provides a clear and comprehensive description of the field of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations.The use of gene transfer is exponentially growing in the scientific and medical communities for day-to-day cell biology experiments and swift development of revolutionary gene therapy strategies. In this advanced textbook, more than 25 leading scientists, world-renowned in their respective fields, come together to provide a clear and comprehensive description of gene delivery, gene therapy and genetic pharmacology.This educational introduction to the main gene transfer vectors and selected therapeutic applications provides the background material needed to further explore the subject as well as relevant research literature. It will thus be invaluable to Master, PhD or MD students, post-doctoral scientists or medical doctors, as well as any scientist wishing to deliver a gene or synthetic nucleotide, or develop a gene therapy strategy. Furthermore, the textbook's simple and synthetic content will be of value to any reader interested in the biological and medical revolution derived from the elucidation of the human genome.

Download or read book Cystic Fibrosis written by Margarida D. Amaral and published by . This book was released on 2011 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Breath from Salt written by Bijal P. Trivedi and published by BenBella Books. This book was released on 2020-09-08 with total page 744 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.