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.

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/.

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.

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.

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/.

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.

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.

Although sickle cell anemia was the first molecular disease to be identified, its complex and fascinating pathophysiology is still not fully understood. A single mutation in the beta-globin gene incurs numerous molecular and cellular mechanisms that contribute to the plethora of symptoms associated with the disease. Our knowledge regarding sickle cell disease mechanisms, while still not complete, has broadened considerably over the last decades. Sickle Cell Anemia: From Basic Science to Clinical Practice aims to provide an update on our current understanding of the disease’s pathophysiology and use this information as a basis to discuss its manifestations in childhood and adulthood. Current therapies and prospects for the development of new approaches for the management of the disease are also covered.

Epigenetics is the study of heritable changes in gene function that do not involve changes in the DNA sequence. These changes, consisting principally of DNA methylation, histone modifications, and non-coding RNAs, maintain or modulate the initial impact of regulatory factors that recognize and associate with particular genomic sequences. Epigenetic modifications are manifest in all aspects of normal cellular differentiation and function, but they can also have damaging effects that result in pathologies such as cancer. Research is continuously uncovering the role of epigenetics in a variety of human disorders, providing new avenues for therapeutic interventions and advances in regenerative medicine. This book's primary goal is to establish a framework that can be used to understand the basis of epigenetic regulation and to appreciate both its derivation from genetics and interdependence with genetic mechanisms. A further aim is to highlight the role played by the three-dimensional organization of the genetic material itself (the complex of DNA, histones and non-histone proteins referred to as chromatin), and its distribution within a functionally compartmentalized nucleus. This architectural organization of the genome plays a major role in the subsequent retrieval, interpretation, and execution of both genetic and epigenetic information.

The study of stem cell biology is under intensive investigations. Because stem cells have the unique capability to self-renew and differentiate into one or several cell types, they play a critical role in development, tissue homeostasis and regeneration. Stem cells also constitute promising cell candidates for cell therapy.The aim of this book is to provide an accurate knowledge on stem cell biology and regenerative medicine. This book will cover many topics in the field and is based on seminars given by recognized scientists involved the international master program on stem cell biology at the University Pierre and Marie Curie (UPMC) in Paris.