Emerging Therapeutic Approaches for Cystic Fibrosis

Emerging Therapeutic Approaches for Cystic Fibrosis PDF Author: Miquéias Lopes-Pacheco
Publisher: Frontiers Media SA
ISBN: 2889633675
Category :
Languages : en
Pages : 281

Book Description


The Cystic Fibrosis Transmembrane Conductance Regulator

The Cystic Fibrosis Transmembrane Conductance Regulator PDF Author: Kevin L. Kirk
Publisher: Springer Science & Business Media
ISBN: 9780306478376
Category : Science
Languages : en
Pages : 236

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.

Cystic Fibrosis

Cystic Fibrosis PDF Author: Prashant Mohite
Publisher: BoD – Books on Demand
ISBN: 1838810730
Category : Medical
Languages : en
Pages : 126

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.

Exploring Novel Clinical Trial Designs for Gene-Based Therapies

Exploring Novel Clinical Trial Designs for Gene-Based Therapies PDF Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309672988
Category : Medical
Languages : en
Pages : 127

Book Description
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.

Gene Therapy Technologies, Applications and Regulations

Gene Therapy Technologies, Applications and Regulations PDF Author: Anthony Meager
Publisher: John Wiley & Sons
ISBN:
Category : Medical
Languages : en
Pages : 450

Book Description
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 in the Light of New Research

Cystic Fibrosis in the Light of New Research PDF Author: Dennis Wat
Publisher: BoD – Books on Demand
ISBN: 9535121529
Category : Medical
Languages : en
Pages : 394

Book Description
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.

Immunopharmacology

Immunopharmacology PDF Author: Manzoor M. Khan
Publisher: Springer Science & Business Media
ISBN: 0387779760
Category : Medical
Languages : en
Pages : 275

Book Description
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.

John Ploughman's Talk

John Ploughman's Talk PDF Author: Charles Haddon Spurgeon
Publisher:
ISBN:
Category : Aphorisms and apothegms
Languages : en
Pages : 264

Book Description


Rare Diseases and Orphan Products

Rare Diseases and Orphan Products PDF Author: Institute of Medicine
Publisher: National Academies Press
ISBN: 0309158060
Category : Medical
Languages : en
Pages : 442

Book Description
Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

Cystic Fibrosis

Cystic Fibrosis PDF Author: David M. Orenstein
Publisher: Lippincott Williams & Wilkins
ISBN: 1451154380
Category : Medical
Languages : en
Pages : 788

Book Description
This one-of-a-kind guide offers easy-to-understand explanations, advice, and management options for patients or parents of patients with cystic fibrosis. The book explains the disease process, outlines the fundamentals of diagnosing and screening, and addresses the challenges of treatment for those living with CF. As one reviewer said, this book “is the only complete answer book for everyone living with the disease. It is an indispensable resource for families of children with CF, adolescent and adult patients, and physicians, nurses, respiratory therapists, and social workers involved in the care of CF patients.”