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Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease.
- Source :
- Annals of the New York Academy of Sciences; Aug2010, Vol. 1202 Issue 1, p59-63, 5p
- Publication Year :
- 2010
-
Abstract
- Effective gene therapy for hemoglobinopathies will require high numbers of autologous gene-engineered hematopoetic stem cells to be reintroduced into the patients. Stem cell mobilization using G-CSF is the most convenient and effective approach to achieve this goal, but it can have severe side effects in sickle cell anemia and be potentially harmful in the case of severe thalassemia. Hence, the optimal way of collection of hematopoetic stem cells from patients with thalassemia and sickle cell disease needs to be determined. In this paper, we review the possible risks of G-CSF mobilization in hemoglobinopathies and we outline the approaches used in an on-going clinical trial in which pretreatment with hydroxyurea is used to reduce potential risks of G-CSF administration to patients with severe beta thalassemia. [ABSTRACT FROM AUTHOR]
- Subjects :
- HEMATOPOIETIC stem cells
GENE therapy
SICKLE cell anemia
CLINICAL trials
HYDROXYUREA
Subjects
Details
- Language :
- English
- ISSN :
- 00778923
- Volume :
- 1202
- Issue :
- 1
- Database :
- Complementary Index
- Journal :
- Annals of the New York Academy of Sciences
- Publication Type :
- Academic Journal
- Accession number :
- 52649251
- Full Text :
- https://doi.org/10.1111/j.1749-6632.2010.05576.x