The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia
Joint Authors
Papanikolaou, Eleni
Drakopoulou, Ekati
Anagnou, Nicholas P.
Source
Issue
Vol. 2011, Issue 2011 (31 Dec. 2011), pp.1-10, 10 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2011-11-13
Country of Publication
Egypt
No. of Pages
10
Main Subjects
Natural & Life Sciences (Multidisciplinary)
Abstract EN
β-thalassemia is characterized by reduced or absence of β-globin production, resulting in anemia.
Current therapies include blood transfusion combined with iron chelation.
BM transplantation, although curative, is restricted by the matched donor limitation.
Gene therapy, on the other hand, is promising, and its success lies primarily on designing efficient globin vectors that can effectively and stably transduce HSCs.
The major breakthrough in β-thalassemia gene therapy occurred a decade ago with the development of globin LVs.
Since then, researchers focused on designing efficient and safe vectors, which can successfully deliver the therapeutic transgene, demonstrating no insertional mutagenesis.
Furthermore, as human HSCs have intrinsic barriers to HIV-1 infection, attention is drawn towards their ex vivo manipulation, aiming to achieve higher yield of genetically modified HSCs.
This paper presents the current status of gene therapy for β-thalassemia, its success and limitations, and the novel promising strategies available involving the therapeutic role of HSCs.
American Psychological Association (APA)
Drakopoulou, Ekati& Papanikolaou, Eleni& Anagnou, Nicholas P.. 2011. The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia. Stem Cells International،Vol. 2011, no. 2011, pp.1-10.
https://search.emarefa.net/detail/BIM-513957
Modern Language Association (MLA)
Drakopoulou, Ekati…[et al.]. The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia. Stem Cells International No. 2011 (2011), pp.1-10.
https://search.emarefa.net/detail/BIM-513957
American Medical Association (AMA)
Drakopoulou, Ekati& Papanikolaou, Eleni& Anagnou, Nicholas P.. The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia. Stem Cells International. 2011. Vol. 2011, no. 2011, pp.1-10.
https://search.emarefa.net/detail/BIM-513957
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-513957