Download or read book Gene and Cell Therapies for Beta Globinopathies written by Punam Malik and published by Springer. This book was released on 2017-11-09 with total page 254 pages. Available in PDF, EPUB and Kindle. Book excerpt: 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.

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 New Insights for the Healthcare Professional 2011 Edition written by and published by ScholarlyEditions. This book was released on 2012-01-09 with total page 59 pages. Available in PDF, EPUB and Kindle. Book excerpt: Sickle Cell Anemia: New Insights for the Healthcare Professional: 2011 Edition is a ScholarlyBrief™ that delivers timely, authoritative, comprehensive, and specialized information about Sickle Cell Anemia in a concise format. The editors have built Sickle Cell Anemia: New Insights for the Healthcare Professional: 2011 Edition on the vast information databases of ScholarlyNews.™ You can expect the information about Sickle Cell Anemia in this eBook to be deeper than what you can access anywhere else, as well as consistently reliable, authoritative, informed, and relevant. The content of Sickle Cell Anemia: New Insights for the Healthcare Professional: 2011 Edition has been produced by the world’s leading scientists, engineers, analysts, research institutions, and companies. All of the content is from peer-reviewed sources, and all of it is written, assembled, and edited by the editors at ScholarlyEditions™ and available exclusively from us. You now have a source you can cite with authority, confidence, and credibility. More information is available at http://www.ScholarlyEditions.com/.

Download or read book Advances in Pediatrics E Book 2020 written by and published by Elsevier Health Sciences. This book was released on 2020-08-07 with total page pages. Available in PDF, EPUB and Kindle. Book excerpt: Each year, Advances in Pediatrics focuses on providing current clinical information on important topics in pediatrics. Dr. Carol Berkowitz and her editorial board, comprised of top experts in the field, have assembled authors to provide updates on the following topics: Evaluation and Management of Febrile Infants; Pediatric Emergency Medicine and Ultrasonography; The Patient-Centered Pediatric Emergency Department; Health Considerations of Refuge and Immigrant Children; Management of scoliosis; Health and Wellness for LGBTQ Youth; Sexually exploited children: recognizing and addressing; Movement disorders in children; Childhood trauma management in primary care; Feeding issues in young children; Physician Well-being and Burnout; New Molecular Methods for Diagnosing Infectious Diseases; Parental refusal: treatments, procedures and vaccines; Pediatric oncology in the ICU setting; Diaphragmatic hernia: Management and Outcomes; and Global Health and Pediatric Education: Opportunities and Challenges. Readers will come away with the clinical information that supplements their professional knowledge so they can make informed clinical decisions that improve patient outcomes.

Download or read book Medical and Surgical Complications of Sickle Cell Anemia written by Ahmed Al-Salem and published by Springer. This book was released on 2015-12-14 with total page 352 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book is a wide-ranging guide to the diagnosis and management of the numerous medical and surgical complications that may arise in patients with sickle cell anemia. After introductory chapters on the genetics, pathophysiology, clinical features, and variants of sickle cell anemia, the complications observed in different parts of the body are addressed in a series of well-illustrated chapters. The coverage includes splenic, hepatobiliary, musculoskeletal, gastrointestinal, ophthalmological, cardio- and cerebrovascular, and renal complications, as well as acute chest syndrome, leg ulcers, hand and foot syndrome, acute appendicitis, and priapism. Treatment-oriented chapters consider perioperative management, blood transfusion therapy, hydroxyurea treatment, hematopoietic stem cell transplantation, and emerging strategies. The book is clearly written in a distinctive bullet point format for ease of reference and emphasizes especially aspects of practical significance. It will be of value for hematologists, general surgeons, internists, pediatricians, pediatric surgeons, fellows, residents, medical students, and nurses.

Download or read book Clinical Hematology and Fundamentals of Hemostasis written by Denise Harmening and published by F.A. Davis. This book was released on 2024-02-01 with total page 2581 pages. Available in PDF, EPUB and Kindle. Book excerpt: A full-color text, lab manual, & atlas—all in one! Here are all the tools medical laboratory science students need to master the principles of hematology and the fundamentals of hemostasis. Author Denise M. Harmening has curated contributions from a team of expert educators and clinicians. With support from her Associate Editor LeAnne Hutson, she brings you comprehensive, yet focused coverage that prepares you for the real world in which you will practice. Begin with an introduction to clinical hematology and the anemias, and then progress through white blood cell disorders and hemostasis to thrombosis and laboratory methods. Find step-by-step laboratory procedures and critical-thinking cases online at FADavis.com for easy access anytime, anywhere.

Download or read book Precision Medicine CRISPR and Genome Engineering written by Stephen H. Tsang and published by Springer. This book was released on 2017-11-11 with total page 180 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book presents descriptive overviews of gene editing strategies across multiple species while also offering in-depth insight on complex cases of application in the field of tissue engineering and regenerative medicine. Chapters feature contributions from leaders in stem cell therapy and biology, providing a comprehensive view of the application of gene therapy in numerous fields with an emphasis on ophthalmology, stem cells, and agriculture. The book also highlights recent major technological advances, including ZFN, TALEN, and CRISPR. Precision Medicine, CRISPR, and Genome Engineering is part of the highly successful Advances in Experimental Medicine and Biology series. It is an indispensable resource for researchers and students in genetics as well as clinicians.

Download or read book Red Book Atlas of Pediatric Infectious Diseases written by American Academy of Pediatrics and published by . This book was released on 2007 with total page 440 pages. Available in PDF, EPUB and Kindle. Book excerpt: Based on key content from Red Book: 2006 Report of the Committee on Infectious Diseases, 27th Edition, the new Red Bookr Atlas is a useful quick reference tool for the clinical diagnosis and treatment of more than 75 of the most commonly seen pediatric infectious diseases. Includes more than 500 full-color images adjacent to concise diagnostic and treatment guidelines. Essential information on each condition is presented in the precise sequence needed in the clinical setting: Clinical manifestations, Etiology, Epidemiology, Incubation period, Diagnostic tests, Treatment

Download or read book Lentivirus New Insights for the Healthcare Professional 2011 Edition written by and published by ScholarlyEditions. This book was released on 2012-01-09 with total page 28 pages. Available in PDF, EPUB and Kindle. Book excerpt: Lentivirus: New Insights for the Healthcare Professional: 2011 Edition is a ScholarlyPaper™ that delivers timely, authoritative, and intensively focused information about Lentivirus in a compact format. The editors have built Lentivirus: New Insights for the Healthcare Professional: 2011 Edition on the vast information databases of ScholarlyNews.™ You can expect the information about Lentivirus in this eBook to be deeper than what you can access anywhere else, as well as consistently reliable, authoritative, informed, and relevant. The content of Lentivirus: New Insights for the Healthcare Professional: 2011 Edition has been produced by the world’s leading scientists, engineers, analysts, research institutions, and companies. All of the content is from peer-reviewed sources, and all of it is written, assembled, and edited by the editors at ScholarlyEditions™ and available exclusively from us. You now have a source you can cite with authority, confidence, and credibility. More information is available at http://www.ScholarlyEditions.com/.

Download or read book Emerging Therapies Targeting the Pathophysiology of Sickle Cell Disease An Issue of Hematology Oncology Clinics written by Elliot Vichinsky and published by Elsevier Health Sciences. This book was released on 2014-04-28 with total page 281 pages. Available in PDF, EPUB and Kindle. Book excerpt: This issue of Hematology/Oncology Clinics, guest edited by Dr. Elliott Vichinsky, is devoted to Sickle Cell Disease, and focuses on pathophysiology of hemoglobinopathies, therapeutic targets, and new approaches to correcting ineffective erythropoiesis and iron dysregulation. Articles in this issue include Polymerization and red cell membrane changes; Overview on reperfusion injury in the pathophysiology of SCD; Regulation of ineffective erythropoiesis in iron metabolism; Altering oxygen affinity; Cellular adhesion and the endothelium; Arginine therapy; Role of the hemostatic system on SCD pathophysiology and potential therapeutics; Adenosine signaling and novel therapies; New approaches to correcting ineffective erythropoiesis and iron dysregulation; New approaches to correcting ineffective erythropoiesis and iron dysregulation; Fetal hemoglobin induction; Gene therapy for hemoglobinopathies; and Oxidative injury and the role of antioxidant therapy.

Download or read book Transitioning Care from Pediatric to Adult Pulmonology written by Cynthia D. Brown and published by Springer Nature. This book was released on 2021-04-28 with total page 169 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book addresses an unmet need in the care of adolescents and young adults with lung disease. The increasing survival of young adults with childhood-onset pulmonary conditions is a testament to major advances in treatments and health care delivery. With the increase in survival of children with chronic respiratory conditions, there is a need for formalized transition programs to integrate adolescents and young adults into the adult model of care. This book helps fill gaps in knowledge to best achieve that initiative. This book takes a comprehensive approach to transition care in pulmonary medicine by satisfying the following objectives: 1) Understand the barriers and developmental challenges in the transition from pediatric to adult care for patients with chronic childhood-onset pulmonary disease; 2) Learn about successful evidence based transition models in pulmonary disease populations, focusing on key process and outcome measures for success; and 3) Develop knowledge to design, implement, and measure a transition program based on evidence and expert opinion. In the first section, the book outlines general principles of transition care that are applicable to all patients regardless of underlying disease process and describe best practices for performing necessary research in transition care. In the second section, the book explores psychosocial factors known to play a role in affecting transition outcomes, including parental support, psychological development, and socioeconomic factors. In the final section, transition outcomes and best practices in specific respiratory diseases are outlined. With a focus on populations with chronic pulmonary disease, this book highlights the challenges and barriers of transition, reviews model systems to understand the essential components of a transition program, and provides evidence-based information to navigate these barriers and achieve successful outcomes during transition to adulthood. This is an ideal guide for pediatric and adult pulmonary providers caring for patients who are transition age, as well as multidisciplinary care team members who work with these providers in care models on transition projects to improve the transition process.

Download or read book Regulatory Aspects of Gene Therapy and Cell Therapy Products written by Maria Cristina Galli and published by Springer. This book was released on 2015-09-15 with total page 235 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Download or read book Thalassemia An Issue of Hematology Oncology Clinics of North America written by Ali Taher and published by Elsevier Health Sciences. This book was released on 2018-03-07 with total page 209 pages. Available in PDF, EPUB and Kindle. Book excerpt: This issue of Hematology/Oncology Clinics, edited by Dr. Ali Taher, focuses on Thalassemia. Topics include, but are not limited to, Molecular basis and genetic modifiers; Evolving spectrum of epidemiology; Clinical classification; Ineffective erythropoiesis, anemia and iron overload; Hypercoagulability and vascular disease; Clinical complications and their management; Transfusion and iron chelation therapy; Hematopoietic Stem Cell Transplantation in Thalassemia; Gene therapy and genome editing; Emerging therapies; Quality of life; Advances in understanding pathophysiology and treatment of fertility, pregnancy, and prenatal diagnosis in Thalassemia; and MRI for iron overload.

Download or read book Management of Chronic Kidney Disease written by Mustafa Arıcı and published by Springer Nature. This book was released on with total page 594 pages. Available in PDF, EPUB and Kindle. Book excerpt:

Download or read book Evidence Based Critical Care written by Paul Ellis Marik and published by Springer. This book was released on 2014-12-08 with total page 836 pages. Available in PDF, EPUB and Kindle. Book excerpt: This is the premier evidence-based textbook in critical care medicine. The Third Edition features updated and revised chapters, numerous new references, streamlined content, and new chapters on key topics such as the new paradigm in critical care medicine, cardiac output monitoring, surgical optimization, vital signs, and arterial blood gas analysis. The book maintains the author’s trademark humor and engaging writing style and is suitable for a broad and diverse audience of medical students, residents, fellows, physicians, nurses, and respiratory therapists who seek the latest and best evidence in critical care. From reviews of previous editions: “This is an excellent introduction to the concept of evidence-based medicine...The writing is clear, logical, and highly organized, which makes for fast and enjoyable reading. I believe this book will get daily use in most intensive care units, by a wide range of readers.” –Respiratory Care “This is one of the most comprehensive handbooks on critical care medicine with a strong emphasis on evidence base...Overall, this book should be useful for junior doctors or intensive care trainees who are starting their term in an intensive care unit.” –Anaesthesia and Intensive Care

Download or read book Prenatal and Preimplantation Diagnosis written by Joann Paley Galst and published by Springer. This book was released on 2015-08-26 with total page 356 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book aims to expand the awareness and understanding of the emotional sequelae of prenatal/preimplantation diagnosis, prenatal decision-making, pregnancy interruption for fetal anomaly, multifetal reduction for high-order multifetal pregnancies and preimplantation choices involving the selection of embryos. Featuring a multi-disciplinary approach, it examines prenatal and preimplantation diagnosis from medical, legal, ethical and psychosocial perspectives. Prenatal and Preimplantation Diagnosis is an excellent resource for obstetricians, reproductive endocrinologists, clinical geneticists, genetic counselors and mental health professionals seeking to better support patients faced with difficult choices.

Download or read book Gene Therapy for HIV and Chronic Infections written by Ben Berkhout and published by Springer. This book was released on 2015-03-10 with total page 246 pages. Available in PDF, EPUB and Kindle. Book excerpt: This book centers on gene therapy and gene transfer approaches to prevent or treat chronic virus infections. The main focus is on the Big Three: human immunodeficiency virus (HIV-1), hepatitis B virus (HBV) and hepatitis C virus (HCV). Ample anti-HIV drugs are currently available in the clinic and the development of an effective combination therapy has dramatically improved the lifespan and quality of life of infected individuals. A similar trend can already be recognized for HBV and HCV: the development of multiple (directly acting) antiviral drugs and plans to control or even cure the infection. However, approaches that help prevent infection, or which provide long-lasting treatment (such as a cure) remain important goals. Immunization through gene transfer vehicles encoding immunogenic viral proteins shows promise in preventing infections with complex, highly variable, viruses such as HIV-1 or HCV. Gene therapy applications for virus infections have been discussed since the early 1990’s. Whereas a true cure seems difficult to achieve for HIV-1 due to its intrinsic property to deposit its genome into that of the host, such attempts may be within reach for HCV where spontaneous viral clearance occurs in a small percentage of the infected individuals. The prospect of original gene therapy approaches may provide alternative ways to reach the same endpoint by, for example, silencing of CCR5 expression post-transcriptionally. Many alternative antiviral strategies have been developed based on a variety of novel molecular methods: e.g. ribozymes. Some studies have progressed towards pre-clinical animal models and a few antiviral gene therapies have progressed towards clinical trials. This book provides an overview of this rapidly progressing field, while focusing on the interface of gene therapy and immunology/vaccinology.