La thérapie génique dans le cadre de la B-thalassémie PDF Download
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Author: Aurélie Quintal
Publisher:
ISBN:
Category :
Languages : fr
Pages : 206
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Book Description
Author: Aurélie Quintal
Publisher:
ISBN:
Category :
Languages : fr
Pages : 206
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Book Description
Author: Punam Malik
Publisher: Springer
ISBN: 1493972995
Category : Medical
Languages : en
Pages : 254
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Book Description
Hemoglobin defects, specifically sickle cell disease & thalassemia, combined, constitute the most common monogenic disorders in the world. In fact, nearly 2% of the world’s population carries a globin gene mutation. The transfer of the corrective globin gene through the HSC compartment by allogeneic HSC transplantation (HSCT) has already proven curative in both SCD and thalassemia patients, and provides the proof of concept that genetic manipulation of the defective organ might be equally therapeutic. However, procedural toxicities and the requirement of an HLA-matched sibling donor limit this approach to a fraction of affected individuals. The editors review the progress & the state of the field in HSCT for hemoglobinopathies & shed light on the major changes expected in the next decade. Although allogeneic HSCT is a curative option, it is limited by the availability of matched donors, which are often available only to 15-20% of patients. An alternative to allogeneic HS CT is genetic correction of autologous HSCs, to overcome donor availability & immune side effects. This Book reviews the progress made on additive gene therapy approaches & the current state of the field. Finally, targeted genetic correction is emerging as a novel therapeutic strategy in the hemoglobinopathies. Although ideal, the inefficiency of targeted correction was rate limiting for translation of this technology to the clinic. With advancements in zinc finger nucleases and TALE endonuclease mediated targeted correction, correction frequencies in hematopoietic stem cells is now reaching levels that may become clinically relevant. Furthermore, the ability to generate autologous embryonic stem cell like cells from primary somatic cells (skin fibroblasts or hematopoietic cells) of the affected individual has allowed for the potential application of genetic correction strategies.This Book reviews upcoming genetic strategies to reactivate fetal hemoglobin production and research advances.
Author: Angelo A. Licata
Publisher: Springer Science & Business Media
ISBN: 1441955496
Category : Medical
Languages : en
Pages : 400
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Book Description
For practitioners at all stages of experience, diseases of the parathyroid glands are not easily deciphered. There are many subtleties to its pathology and diagnosis. Today, the wealth of new information from molecular medicine makes it very challenging for the individual practitioner to have a complete picture of these entities. Diseases of the Parathyroid Glands addresses these issues by offering a concise, user-friendly text that provides state-of-the-art background on mineral physiology and its regulation and couples this to a variety of clinical topics of parathyroid gland pathology. Introductory chapters cover calcium regulation and parathyroid gland physiology, drawing upon many of the new aspects of glandular control mechanisms beyond the classical ones associated with calcium alone. The majority of the text covers clinical problems. Although most chapters address problems in adult medicine, three separate chapters are devoted to pediatric problems of hyper- and hypoparathyroidsm and parathormone resistance states. In addition, there are several chapters on less common but equally challenging problems such as parathyroid cancer, calciphylaxis, cystic lesions of the glands, and ectopic glandular disease. The final chapters detail the techniques of parathyroid gland imaging and surgical treatment. Practical and an invaluable addition to the literature, Diseases of the Parathyroid Glands is an indispensable reference for anyone interested in parathyroid disease.
Author: Izuho Hatada
Publisher: Humana
ISBN: 9781071630150
Category : Science
Languages : en
Pages : 0
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Book Description
This second edition provides new and updated protocols that can be used for generation of knockout animals. Chapters guide the reader through basic protocols for three genome editing technologies, target design tools, and specific protocols for each animal. Written in the successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible protocols, and notes on troubleshooting and avoiding known pitfalls. Authoritative and cutting-edge, Genome Editing in Animals: Methods and Protocols, Second Edition aims to be a useful practical guide to researches to help further their study in this field.
Author: Giuseppe D'Amico
Publisher: S. Karger AG (Switzerland)
ISBN:
Category : Medical
Languages : en
Pages : 328
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Book Description
Author: Roderick N. M. MacSween
Publisher:
ISBN: 9780443061813
Category : Medical
Languages : en
Pages : 982
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Book Description
Here is the new edition of the definitive reference work on liver pathology. Comprehensive, authoritative and superbly illustrated, this book leads the field, with editors and contributors who represent a "who's who" of hepatopathology. The fourth edition is an invaluable bench-side reference and research tool for the day-to-day diagnosis of the wide range of appearances of the liver resulting from infection, tumours and tumour-like lesions or damage caused by drugs or toxins
Author: Samir K. Ballas
Publisher: Lippincott Williams & Wilkins
ISBN: 1496331834
Category : Medical
Languages : en
Pages : 1004
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Book Description
Sickle Cell Pain is a panoramic, in-depth exploration of every scientific, human, and social dimension of this cruel disease. This comprehensive, definitive work is unique in that it is the only book devoted to sickle cell pain, as opposed to general aspects of the disease. The 752-page book links sickle cell pain to basic, clinical, and translational research, addressing various aspects of sickle pain from molecular biology to the psychosocial aspects of the disease. Supplemented with patient narratives, case studies, and visual art, Sickle Cell Pain’s scientific rigor extends through its discussion of analgesic pharmacology, including abuse-deterrent formulations. The book also addresses in great detail inequities in access to care, stereotyping and stigmatization of patients, the implications of rapidly evolving models of care, and recent legislation and litigation and their consequences.
Author: Ingolf Bernhardt
Publisher: Springer Science & Business Media
ISBN: 3662051818
Category : Science
Languages : en
Pages : 761
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Book Description
51 worldwide leading experts in the field of erythrocyte research contributed to this first book on transport processes in red blood cells. It explains the latest findings on the basis of well-established principles, in an accessibly structured and carefully organized compilation.
Author: Erik Parens
Publisher: JHU Press
ISBN: 9780801882241
Category : Medical
Languages : en
Pages : 388
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Book Description
Wrestling with Behavioral Genetics brings together an interdisciplinary group of contributors -- geneticists, humanists, social scientists, lawyers, and journalists -- to discuss the ethical and social implications of behavioral genetics research. The essays give readers the necessary tools to critically analyze the findings of behavioral geneticists, explore competing interpretations of the ethical and social implications of those findings, and engage in a productive public conversation about them. "What sets this collection apart from others is the way that contributions from a diverse authorship are integrated to form a coherent whole... Doubtless this book will soon become a classic within behavioral genetics and compulsory reading for the non-specialist seeking to understand the basic scientific, social, and ethical issues within the field." -- American Journal of Bioethics "Informative, provocative, and challenging, this book is a must-read for anyone seeking to understand this emerging field." -- Social Theory and Practice "Promoting public conversation about behavioral genetics will be increasingly pertinent to creating enlightened, fair, and representative public policy... The 'wrestling' will go on for some time to come." -- New England Journal of Medicine "This volume presents a fair and honest treatment of the field that is both cautious at times and also optimistic and hopeful." -- Metapsychology Erik Parens is a senior research scholar at the Hastings Center and a visiting professor in the Science, Technology, and Society Program at Sarah Lawrence College. Audrey R. Chapman is a professor of community medicine and Healey Chair in Medical Humanities and Bioethics at the University of Connecticut School of Medicine. Nancy Press is a professor at the School of Nursing and the Department of Public Health at the School of Medicine, Oregon Health and Science University.
Author: Hubert M. Blalock Jr.
Publisher: UNC Press Books
ISBN: 0807873020
Category : Philosophy
Languages : en
Pages : 214
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Book Description
Taking an exploratory rather than a dogmatic approach to the problem, this book pulls together materials bearing on casual inference that are widely scattered in the philosophical, statistical, and social science literature. It is written in nonmathematical terms, and it is imaginative and sophisticated from both a theoretical and a statistical point of view. Originally published in 1964. A UNC Press Enduring Edition -- UNC Press Enduring Editions use the latest in digital technology to make available again books from our distinguished backlist that were previously out of print. These editions are published unaltered from the original, and are presented in affordable paperback formats, bringing readers both historical and cultural value.
