Gene therapy for β-thalassaemia: the continuing challenge

Evangelia Yannaki; David W. Emery; George Stamatoyannopoulos

doi:10.1017/S1462399410001626

Gene therapy for β-thalassaemia: the continuing challenge

Published online by Cambridge University Press: 01 October 2010

Evangelia Yannaki ,

David W. Emery and

George Stamatoyannopoulos

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Evangelia Yannaki*: Affiliation:
Gene and Cell Therapy Center, Hematology Department-BMT Unit, George Papanicolaou Hospital, Thessaloniki, Greece.
David W. Emery: Affiliation:
Department of Medicine, Division of Medical Genetics, University of Washington, Seattle, WA, USA. Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA.
George Stamatoyannopoulos: Affiliation:
Department of Medicine, Division of Medical Genetics, University of Washington, Seattle, WA, USA. Department of Genome Sciences, University of Washington, Seattle, WA, USA.
*: *Corresponding author: Evangelia Yannaki, G. Papanicolaou Hospital, Gene and Cell Therapy Center, Hematology-BMT Unit, Thessaloniki 57010, Greece. E-mail: eyannaki@u.washington.edu

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Abstract

The β-thalassaemias are inherited anaemias that form the most common class of monogenic disorders in the world. Treatment options are limited, with allogeneic haematopoietic stem cell transplantation offering the only hope for lifelong cure. However, this option is not available for many patients as a result of either the lack of compatible donors or the increased risk of transplant-related mortality in subjects with organ damage resulting from accumulated iron. The paucity of alternative treatments for patients that fall into either of these categories has led to the development of a revolutionary treatment strategy based on gene therapy. This approach involves replacing allogeneic stem cell transplantation with the transfer of normal globin genes into patient-derived, autologous haematopoietic stem cells. This highly attractive strategy offers several advantages, including bypassing the need for allogeneic donors and the immunosuppression required to achieve engraftment of the transplanted cells and to eliminate the risk of donor-related graft-versus-host disease. This review discusses the many advances that have been made towards this endeavour as well as the hurdles that must still be overcome before gene therapy for β-thalassaemia, as well as many other gene therapy applications, can be widely applied in the clinic.

Type: Review Article
Information: Expert Reviews in Molecular Medicine , Volume 12 , January 2010 , e31

DOI: https://doi.org/10.1017/S1462399410001626 [Opens in a new window]
Copyright: Copyright © Cambridge University Press 2010

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Gene therapy for β-thalassaemia: the continuing challenge

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Gene therapy for β-thalassaemia: the continuing challenge

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