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Gene therapy

Targeting β-thalassaemia

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.

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Figure 1: Gene-therapy procedure.

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

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