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55293 Posts in 5909 Topics by 6206 Members
Latest Member: jammirati

A message for all  parents who are thals. Keeping your iron load under control is an absolute obligation to your children.
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Author Topic: B Thalassaemia patient became transfusion free following Gene Therapy  (Read 3846 times)
Akita
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« on: November 10, 2010, 01:15:22 PM »

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

Citation from the Abstract>

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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