This book is the story of an inherited blood disease -- Cooley's anemia or beta thalassemia. Cooley's anemia is a severe and potentially fatal anemia that affects millions of people worldwide. Written by a world-recognized expert on the disease who has contributed greatly to the scientific understanding of the pathophysiology of this human hemoglobin disorder, the book describes the emotional and medical impact of the disease on patients and their families. It also summarizes the research on the disease at the level of the genes, DNA, RNA, and proteins. In addition, it also includes chapters on current therapy and future approaches to the disease, such as gene therapy, stem cell transplantation, and antenatal diagnosis.A multidisciplinary book covering the fields of hematology, pediatrics, medicine, genetics, and molecular biology, the book details how Cooley's anemia serves as a model for understanding other human genetic and acquired diseases such as cancer, neurological disorders, and heart disease. It also provides new insights which may lead to new therapies.
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