Haemoglobin Disorders: Gene Therapy
Published Online: 15 SEP 2009
Copyright © 2001 John Wiley & Sons, Ltd. All rights reserved.
How to Cite
Persons, D. A. and Nienhuis, A. W. 2009. Haemoglobin Disorders: Gene Therapy. eLS.
- Published Online: 15 SEP 2009
The ability to transfer a globin gene into stem cells and achieve regulated expression during erythropoiesis following transplantation of genetically modified autologous cells could provide curative therapy for haemoglobin disorders such as sickle cell anaemia or β-thalassaemia. Success has been achieved in several severe immunodeficiencies in which there is a selective advantage for gene corrected cells. The development of effective globin lentiviral vectors has resulted in resolution of the manifestations of sickle cell anaemia and β-thalassaemia in mouse models of these diseases. However, a higher stem cell transduction efficiency must be achieved in humans than has been achieved in large animal models to date before success in treatment of haemoglobinopathies could be expected. Various strategies are being evaluated in animal models to increase the proportion of genetically modified, haematopoietic cells following infusion of transduced stem cells.
- gene therapy;
- sickle cell disease;
- lentiviral vector