期刊
EXPERT REVIEW OF HEMATOLOGY
卷 6, 期 3, 页码 255-264出版社
TAYLOR & FRANCIS LTD
DOI: 10.1586/EHM.13.24
关键词
beta-thalassemia; erythroid progenitor cells; gene therapy; hemoglobin; high persistency of fetal hemoglobin; sickle cell anemia
类别
资金
- NIH [RO1 HL102449-03]
- Fondazione Telethon Funding Source: Custom
beta-thalassemias are caused by nearly 300 mutations of the beta-globin gene, leading to a low or absent production of adult hemoglobin (HbA). Two major therapeutic approaches have recently been proposed: gene therapy and induction of fetal hemoglobin (HbF) with the objective of achieving clinically relevant levels of Hbs. The objective of this article is to describe the development of therapeutic strategies based on a combination of gene therapy and induction of HbFs. An increase of beta-globin gene expression in beta-thalassemia cells can be achieved by gene therapy, although de novo production of clinically relevant levels of adult Hb may be difficult to obtain. On the other hand, an increased production of HbF is beneficial in beta-thalassemia. The combination of gene therapy and HbF induction appears to be a pertinent strategy to achieve clinically relevant results.
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