Chapter title |
Reactivation of Fetal Hemoglobin for Treating β-Thalassemia and Sickle Cell Disease
|
---|---|
Chapter number | 7 |
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_7 |
Pubmed ID | |
Book ISBNs |
978-1-4939-7297-5, 978-1-4939-7299-9
|
Authors |
Shuaiying Cui, James Douglas Engel, Cui, Shuaiying, Engel, James Douglas |
Abstract |
Reactivation of fetal hemoglobin (HbF) in adult hematopoietic cells has the potential for great clinical benefit in patients bearing deleterious mutations in the β-globin gene, such as β-thalassemia and sickle cell disease (SCD), since increasing the production of HbF can compensate for underproduction of β-globin chains (in β-thalassemia) and it can also disrupt sickle hemoglobin polymerization (in SCD). Thus for the past few decades, concerted efforts have been made to identify an effective way to induce the synthesis of HbF in adult erythroid cells for potential therapeutic relief from the effects of these β-globinopathies. Chemical inducers of HbF as well as a number of transcription factors that are able to reactivate HbF synthesis in vitro and in vivo in adult erythroid cells have been identified. However, there has been only limited success in attempts to manipulate either the drugs or regulatory proteins, and in only a fraction of patients, and there is wide variation in individual response to these drugs or transcription factors. These studies highlight the importance for understanding the molecular mechanisms underlying hemoglobin switching so that future studies can be designed to treat these disorders. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
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Unknown | 32 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Student > Bachelor | 6 | 19% |
Student > Ph. D. Student | 5 | 16% |
Student > Doctoral Student | 2 | 6% |
Lecturer | 2 | 6% |
Student > Master | 2 | 6% |
Other | 3 | 9% |
Unknown | 12 | 38% |
Readers by discipline | Count | As % |
---|---|---|
Biochemistry, Genetics and Molecular Biology | 12 | 38% |
Medicine and Dentistry | 5 | 16% |
Agricultural and Biological Sciences | 2 | 6% |
Neuroscience | 1 | 3% |
Engineering | 1 | 3% |
Other | 0 | 0% |
Unknown | 11 | 34% |