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.
Contents:Patients and Families: Love and Loss; In the Beginning; Nunzio: One of a Kind; Connie Paradiso; A Life on the Run; Amy's Loves; Understanding Thalassemia: The Big Alpha; Human Globin Messages; Finding the Genes; The Ultimate Answers; You Can't Go Home Again; The Best Medicine: Current Care and Future Goals: The Standard of Care; Dedicated Doctors; Human Beta Globin Gene Therapy; Genetic Roulette; Modern Times; Love Unbound; The Last Chapter.
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