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Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease
Author(s) -
Yannaki Evangelia,
Stamatoyannopoulos George
Publication year - 2010
Publication title -
annals of the new york academy of sciences
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.712
H-Index - 248
eISSN - 1749-6632
pISSN - 0077-8923
DOI - 10.1111/j.1749-6632.2010.05576.x
Subject(s) - thalassemia , genetic enhancement , stem cell , beta thalassemia , medicine , haematopoiesis , disease , sickle cell anemia , hemoglobinopathy , hematopoietic stem cell , anemia , immunology , gene , biology , genetics
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.