Download or read book Renaissance Of Sickle Cell Disease Research In The Genome Era written by Betty Pace and published by World Scientific. This book was released on 2007-01-24 with total page 394 pages. Available in PDF, EPUB and Kindle. Book excerpt: The Human Genome Project has spawned a Renaissance of research faced with the daunting expectation of personalized medicine for individuals with sickle cell disease in the Genome Era. This book offers a comprehensive and timeless account of emerging concepts in clinical and basic science research, and community concerns of health disparity to educate professionals, students and the general public about meeting this challenging expectation. Contributions from physicians, research scientists, scientific administrators and community workers make Renaissance of Sickle Cell Disease Research in the Genome Era unique among the catalogue of books on this genetic disorder.Part 1 offers detailed review of the National Heart Lung and Blood Institute's leadership role in funding sickle cell research, as well as developing progressive research initiatives and the predicted impact of the Human Genome Project. Part 2 gives an account of several clinical research perspectives based on the Cooperative Study of Sickle Cell Disease. These include recommendations for newborn screening, pain management, stroke, transfusion therapy and pediatric and adult healthcare. Part 3 offers novel insights into basic science research progress and the impact of the Human Genome Project on the direction of hemoglobinopathy research, including hemoglobin switching, bone marrow transplantation and gene therapy. Part 4 engages the reader in a culture-based discussion of the stigma attached to sickle cell disease in the African American community and the apprehensions about genetic research in this community. It concludes with a global perspective on sickle cell disease from African, European and American experiences. For readers seeking a definitive account of sickle cell disease appropriate for students, researchers and community workers, this collaborative effort is an ideal textbook./a

Download or read book New Developments in Sickle Cell Disease Research written by Paul D. O'Malley and published by Nova Publishers. This book was released on 2006 with total page 266 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Sickle Cell Pain written by Samir K. Ballas and published by Lippincott Williams & Wilkins. This book was released on 2015-06-01 with total page 1004 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Sickle Cell Anemia written by Intsar S. Waked and published by Nova Science Publishers. This book was released on 2016 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Sickle cell disease (SCD), an inherited hemolytic anemia, is associated with multiple acute and chronic complications such as painful vasoocclusive events, cerebral vasculopathy, priapism, and renal or lung disease. These complications are variable and unpredictable, and can be associated with significant morbidity and poor quality of life. This book covers several areas regarding pathology, diagnosis, complications, signs, symptoms and medical treatments. There are few studies in literature on the role of physiotherapy as a resource to prevent and treat locomotor system disorders, respiratory problems and painful crises in SCD individuals. This book highlights the role of physiotherapy in sickle cell anemia. A comprehensive and authoritative monograph, this book will be equally interesting to both established researchers and to graduate students interested in both genetics and the physical therapy field.

Download or read book Inherited Hemoglobin Disorders written by Anjana Munshi and published by BoD – Books on Demand. This book was released on 2015-11-11 with total page 198 pages. Available in PDF, EPUB and Kindle. Book excerpt: The book, Inherited Hemoglobin Disorders, describes the genetic defects of hemoglobins, disease complications, and therapeutic strategies. This book has two distinct sections. The first theme includes seven chapters devoted to the types of hemoglobinopathies, mutation spectrum, diagnostic methods, and disease complications, and the second theme includes three chapters focusing on various treatment strategies. The content of the chapters presented in the book is guided by the knowledge and experience of the contributing authors. This book serves as an important resource and review to the researchers in the field of hemoglobinopathies.

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 The Management of Sickle Cell Disease written by U. S. Department of Health and published by Createspace Independent Publishing Platform. This book was released on 2002 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: #1 Best Seller on Sickle Cell Disease (SCD). Sickle cell disease is a group of blood disorders passed down from parents to children. Sickle cell anemia shortens life expectancy by 30 years via bacterial infections, painful swellings, fever, arthritis, leg ulcers, eye, lung & heart damage. Over 100,000 people, mostly African-Americans, in the United States have sickle cell disease. Over 2 million people have sickle cell trait in America. It is estimated that more than 300,000 children are born each year with SCD around the world. This edition of The Management of Sickle Cell Disease (SCD) is organized into four parts: 1. Diagnosis and Counseling 2. Health Maintenance 3. Treatment of Acute and Chronic Complications 4. Special Topics. The original intent was to incorporate evidence-based medicine into each chapter, but there was variation among evidence-level scales, and some authors felt recommendations could be made, based on accepted practice, without formal trials in this rare disorder. The best evidence still is represented by randomized, controlled trials (RCTs), but variations exist in their design, conduct, endpoints, and analyses. It should be emphasized that selected people enter a trial, and results should apply in practice specifically to populations with the same characteristics as those in the trial. Randomization is used to reduce imbalances between groups, but unexpected factors sometimes may confound analysis or interpretation. In addition, a trial may last only a short period of time, but long-term clinical implications may exist. Another issue is treatment variation, for example, a new pneumococcal vaccine developed after the trial, which has not been tested formally in a sickle cell population. Earlier trial results may be accepted, based on the assumption that the change is small. In some cases, RCTs cannot be done satisfactorily (e.g., for ethical reasons, an insufficient number of patients, or a lack of objective measures for sickle cell "crises"). Thus the bulk of clinical experience in SCD still remains in the moderately strong and weaker categories of evidence. Not everyone has an efficacious outcome in a clinical trial, and the frequency of adverse events, such as with long-term transfusion programs or hematopoietic transplants, might not be considered. Thus, an assessment of benefit-to-risk ratio should enter into translation of evidence levels into practice recommendations. A final issue is that there may be two alternative approaches that are competitive (e.g., transfusions and hydroxyurea). In this case the pros and cons of each course of treatment should be discussed with the patient. This book is B&W copy of the government agency publication.

Download or read book Evidence Based Management of Sickle Cell Disease written by M D George R Buchanan and published by Createspace Independent Publishing Platform. This book was released on 2014-09-09 with total page 0 pages. Available in PDF, EPUB and Kindle. Book excerpt: Sickle cell disease can be severe and disabling. When properly treated, patients live longer and with better quality life. This is a US government publication intended to provide evidence-based guidelines for the care of these patients for the use of all concerned providers as well as patients and family members. This book is available in print here for convenience.

Download or read book Disorders of Hemoglobin written by Martin H. Steinberg and published by Cambridge University Press. This book was released on 2009-08-17 with total page 883 pages. Available in PDF, EPUB and Kindle. Book excerpt: Completely revised new edition of the definitive reference on disorders of hemoglobin.

Download or read book Iron Chelation Therapy written by Chaim Hershko and published by Taylor & Francis US. This book was released on 2002 with total page 290 pages. Available in PDF, EPUB and Kindle. Book excerpt: Within the last few years, iron research has yielded exciting new insights into the understanding of normal iron homeostasis. Such development, and the evolution of improved strategies of Iron Chelating Therapy require better understanding of the pathophysiology of iron toxicity and the mechanism of action of iron chelating drugs. The timeliness of the present volume is underlined by several significant developments in recent years. New insights have been gained into the molecular basis of aberrant iron handling in hereditary disorders and the pathophysiology of iron overload. This volume highlights the impact of long term Iron Celating Therapy using deferoxamine or the new, but controversial oral iron chelator deferiprone based on experience gained by multicenter trails, with special emphasis on survival, morbidity and drug toxicity; it reviews the development of the new and improved orally effective chelators suitable for clinical use in the near future and examines novel strategies of iron chelating treatment for the control of cell proliferation in malignant disease or malaria.

Download or read book Newborn Screening for Sickle Cell Disease and other Haemoglobinopathies written by Stephan Lobitz and published by MDPI. This book was released on 2019-10-07 with total page 160 pages. Available in PDF, EPUB and Kindle. Book excerpt: Newborn Screening for Sickle Cell Disease and other Haemoglobinopathies is a Special Issue of the International Journal of Neonatal Screening. Sickle cell disease is one of the most common inherited blood disorders, with a huge impact on health care systems due to high morbidity and high mortality associated with the undiagnosed disease. Newborn screening helps to make the diagnosis early and to prevent fatal complications and diagnostic odysseys. This book gives an overview of diagnostic standards in newborn screening for sickle cell disease and examples of existing newborn screening programs.

Download or read book Heritable Human Genome Editing written by The Royal Society and published by National Academies Press. This book was released on 2021-01-16 with total page 239 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

Download or read book Advancement of Artificial Intelligence in Healthcare Engineering written by Dilip Singh Sisodia and published by Medical Information Science Reference. This book was released on 2020 with total page 300 pages. Available in PDF, EPUB and Kindle. Book excerpt: "This book explores the possible applications of machine learning, deep learning, soft computing, and evolutionary computing techniques in the design, implementation, and optimization of challenging healthcare engineering solutions"--

Download or read book Molecular Hematology written by Drew Provan and published by John Wiley & Sons. This book was released on 2024-03-05 with total page 452 pages. Available in PDF, EPUB and Kindle. Book excerpt: Provides a core knowledgebase for those with minimal exposure to hematologic molecular biology, fully revised and updated Molecular Hematology brings together the most up-to-date and reliable information on the molecular basis of major hematologic diseases. Edited and authored by leading experts in the field, this volume demonstrates the clinical relevance of molecular biology in the diagnosis and treatment of blood disorders. Concise, accessible chapters address key topics in modern hematology, such as lymphoma genetics, molecular coagulation, thrombophilia, pharmacogenomics, platelet disorders, hemophilia, anemia, and multiple myeloma. Chapters introduce and summarize specific hematologic diseases, explore the pathogenesis of the disorder, highlight new and emerging molecular strategies, and more. Now in its fifth edition, Molecular Hematology is fully revised to incorporate contemporary hematology research and practice. Entirely new chapters cover topics such as CAR-T cell technology, COVID, cancer cell biology, gene therapy and gene editing in clinical practice, as well as the impact of molecular research on the diagnosis and treatment of various malignant hematologic diseases. This edition includes updated suggested readings and a wealth of new full-color charts, graphs, and illustrations throughout. Covers the molecular biology of different blood diseases, their pathogeneses, and current molecular research and therapies Illustrates the impact of molecular research on hematologic investigations and therapeutics Discusses how progress in basic science and therapeutics has improved the diagnosis and care of patients with various hematologic disorders Surveys recent developments in cancer-cell biology, with an emphasis on leukemia and lymphoma Includes access to a companion Digital Edition providing search across the book, downloadable illustrations, and notation tools Molecular Hematology is a must-have resource for established and trainee clinicians, clinical scientists and researchers, medical students, and upper-level undergraduates in the fields of hematology, oncology, and molecular biology.

Download or read book Sickle Cell Disease written by Mark T. Gladwin and published by McGraw Hill Professional. This book was released on 2021-01-05 with total page 715 pages. Available in PDF, EPUB and Kindle. Book excerpt: The most comprehensive, current sickle cell disease resource—for both clinicians and researchers A Doody's Core Title for 2023! The first and only resource of its kind, Sickle Cell Disease examines this blood disorder through both clinical and research lenses. More than 80 dedicated experts in the field present their combined clinical knowledge of basic mechanisms, screening, diagnosis, management, and treatment of myriad complex complications of a single base point mutation in the human genome. Case studies with “How I Treat” authoritative insights provide overviews of common and rare complications, and Key Facts offer at-a-glance high-yield information. Filled with clinical photos, illustrations, numerous original diagrams, and with free updates available online, this unmatched resource covers: Mechanisms of sickle cell disease Historic and current research approaches The latest work in gene therapy and editing Guidelines for patient care, diagnosis, unique cases, and therapies Rare and common complications, including domestic and internationally relevant topics Psychosocial and supportive care The newest standards of therapy and future treatment options in children and adults Cardiopulmonary complications

Download or read book Oversight and Review of Clinical Gene Transfer Protocols written by Institute of Medicine and published by National Academies Press. This book was released on 2014-03-27 with total page 78 pages. Available in PDF, EPUB and Kindle. Book excerpt: Gene transfer research is a rapidly advancing field that involves the introduction of a genetic sequence into a human subject for research or diagnostic purposes. Clinical gene transfer trials are subject to regulation by the U.S. Food and Drug Administration (FDA) at the federal level and to oversight by institutional review boards (IRBs) and institutional biosafety committees (IBCs) at the local level before human subjects can be enrolled. In addition, at present all researchers and institutions funded by the National Institutes of Health (NIH) are required by NIH guidelines to submit human gene transfer protocols for advisory review by the NIH Recombinant DNA Advisory Committee (RAC). Some protocols are then selected for individual review and public discussion. Oversight and Review of Clinical Gene Transfer Protocols provides an assessment of the state of existing gene transfer science and the current regulatory and policy context under which research is investigated. This report assesses whether the current oversight of individual gene transfer protocols by the RAC continues to be necessary and offers recommendations concerning the criteria the NIH should employ to determine whether individual protocols should receive public review. The focus of this report is on the standards the RAC and NIH should use in exercising its oversight function. Oversight and Review of Clinical Gene Transfer Protocols will assist not only the RAC, but also research institutions and the general public with respect to utilizing and improving existing oversight processes.

Download or read book Benign Hematologic Disorders in Children written by Deepak M. Kamat and published by Springer Nature. This book was released on 2020-08-18 with total page 512 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book provides a comprehensive overview of benign hematologic disorders in children. Divided into nine sections, the text reviews common hematologic disorders or conditions that affect children, while providing state-of-the-art information on pathophysiology, diagnosis, treatment, and management strategies. The text begins with a section on hematopoiesis, and the next section covers red blood cell disorders. The following sections provide overviews of platelet disorders, white blood cell disorders, and coagulation disorders. The sixth and seventh sections discuss neonatal hematology and bone marrow failure syndrome. The eighth section reviews supportive care, while the final section covers miscellaneous subjects including pediatric vascular anomalies and complement dysregulation syndromes. Written by experts in the field, Benign Hematologic Disorders in Children: A Clinical Guide is a valuable resource for clinicians and practitioners who treat children afflicted with these disorders.