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Gene therapy

Targeting β-thalassaemia

Nature volume 467, pages 277–278 (2010)Cite this article

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Patients with disorders of the blood protein haemoglobin often depend on lifelong blood transfusions. That could change, given the success of gene therapy in a patient with one such disorder.

β-Thalassaemia is one of several inherited disorders associated with abnormalities in the oxygen-carrying protein haemoglobin. It is caused by mutations in the β-globin chain of haemoglobin that lead to ineffective production of red blood cells and profound anaemia. Patients with β-thalassaemia require regular blood transfusions for life. Chronic transfusions have a significant impact on the quality of life and ultimately shorten life expectancy. As for treating this disorder, until now the only available strategy has been the transplantation of bone-marrow cells, a procedure whose success depends on the availability of suitable donors. A therapy based on genetic correction of a patient's own bone-marrow cells has therefore long been awaited1. On page 318 of this issue, Cavazzana-Calvo et al.2 deliver news of one such success story using gene therapy.

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Figure 1: Gene-therapy procedure.

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Authors and Affiliations

  1. Derek A. Persons is in the Department of Hematology, St Jude Children's Research Hospital, Memphis, Tennessee 38105, USA. derek.persons@stjude.org,

    Derek A. Persons

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  1. Derek A. Persons
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Persons, D. Targeting β-thalassaemia. Nature 467, 277–278 (2010). https://doi.org/10.1038/467277a

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