Chapter title |
Clinical Features of β-Thalassemia and Sickle Cell Disease
|
---|---|
Chapter number | 1 |
Book title |
Gene and Cell Therapies for Beta-Globinopathies
|
Published in |
Advances in experimental medicine and biology, November 2017
|
DOI | 10.1007/978-1-4939-7299-9_1 |
Pubmed ID | |
Book ISBNs |
978-1-4939-7297-5, 978-1-4939-7299-9
|
Authors |
Patrick T. McGann, Alecia C. Nero, Russell E. Ware, McGann, Patrick T., Nero, Alecia C., Ware, Russell E. |
Abstract |
Sickle cell disease (SCD) and β-thalassemia are among the most common inherited diseases, affecting millions of persons globally. It is estimated that 5-7% of the world's population is a carrier of a significant hemoglobin variant. Without early diagnosis followed by initiation of preventative and therapeutic care, both SCD and β-thalassemia result in significant morbidity and early mortality. Despite great strides in the understanding of the molecular basis and pathophysiology of these conditions, the burden of disease remains high, particularly in limited resource settings. Current therapy relies heavily upon the availability and safety of erythrocyte transfusions to treat acute and chronic complications of these conditions, but frequent transfusions results in significant iron overload, as well as challenges from acquired infections and alloimmunization. Hydroxyurea is a highly effective treatment for SCD but less so for β-thalassemia, and does not represent curative therapy. As technology and use of cellular and gene therapies expand, SCD and thalassemia should be among the highest disease priorities. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 69 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Master | 9 | 13% |
Student > Bachelor | 9 | 13% |
Student > Ph. D. Student | 4 | 6% |
Professor > Associate Professor | 3 | 4% |
Student > Postgraduate | 3 | 4% |
Other | 8 | 12% |
Unknown | 33 | 48% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 15 | 22% |
Biochemistry, Genetics and Molecular Biology | 9 | 13% |
Nursing and Health Professions | 2 | 3% |
Agricultural and Biological Sciences | 2 | 3% |
Psychology | 2 | 3% |
Other | 5 | 7% |
Unknown | 34 | 49% |