Innovative therapies for red cell and iron related disorders



Fontenay-aux-Roses, France - Boston, Massachusetts, USA
Philippe Leboulch, M.D.1,2,3 and the LentiGlobin clinical trial study group
1 CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI), Fontenay-aux-Roses 92265, France, 2Inserm U96
2 and University Paris XI, CEA-iMETI, Fontenay-aux-Roses 92265, France,
3 Genetics Division, Brigham & Women’s Hospital and Harvard Medical School, Boston, MA 02115, USA. Dr Philippe Leboulch was unable to attend the meeting due to the volcano ash cloud. This talk was given by Pr Yves Beuzard who is co-author of this work.

Conversion to transfusion independence with Hmga2 activation after lentiviral gene therapy for severe human beta-thalassemia

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