B Thalassaemia patient became transfusion free following Gene Therapy

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Transfusion independence and HMGA2 activation after gene therapy of human β-tha

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Here we show that, 33 months after lentiviral β-globin gene transfer, an adult patient with severe βE/β0-thalassaemia dependent on monthly transfusions since early childhood has become transfusion independent for the past 21 months.

http://www.nature.com/nature/journal/v467/n7313/abs/nature09328.html


 

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