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Gene therapy for beta-thalassaemia

https://doi.org/10.1136/archdischild-2018-315444

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    The list of life-limiting inherited conditions for which radically new gene-based treatments are appearing continues to grow. The latest is β-thalassaemia, which affects over ¼ million people worldwide. The majority of sufferers have a lifelong continuing blood transfusion requirement, with all the complications that come with it, such as iron overload. Those fortunate enough to have a compatible relative can sometimes be ‘cured’ by a bone marrow transplant, but that won’t work for many.

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    • Provenance and peer review Commissioned; internally peer reviewed.