Thalassemia Patients and Friends

Discussion Forums => Announcements => Topic started by: Akita on November 10, 2010, 07:15:22 PM

Title: B Thalassaemia patient became transfusion free following Gene Therapy
Post by: Akita on November 10, 2010, 07:15:22 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