Development and recent progresses of gene therapy for β-thalassemia
β-thalassemias are among the most common inherited monogenic disorders worldwide due to mutations in the β-globin gene that reduce or abolish the production of the β-globin chain resulting in transfusion-dependent chronic anemia. Currently, the only curative treatment is allogeneic hematopoietic ste...
| Main Authors: | , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2014-09-01
|
| Series: | Thalassemia Reports |
| Subjects: | |
| Online Access: | http://www.pagepressjournals.org/index.php/thal/article/view/2925 |