ere beta-thalassemia is living without the need for transfusions over a sustained period of time," said Marina Cavazzana-Calvo, M.D., first co-author of the study and professor of hematology, University of Paris and chief of Cell and Gene Therapy Department, Necker-Enfants Malades Hospital in Paris. Salima Hacein-Bey-Abina, Ph.D., professor of immunology, University of Paris, added, "These results are not only important due to the tremendous medical need that exists for thalassemia patients around the world, but also represents a significant step forward for the field of autologous stem cell therapy as an emerging therapeutic modality."
Dr. Franoise Bernaudin, the clinical hematologist who has followed this patient since early childhood, said, "It is wonderful to see that this young man is for now free of transfusions and injections for iron chelation. He is happy to have a normal life back, and for the first time has a full-time job as a cook in a main restaurant in Paris. We are now even able to bleed him regularly to help remove toxic iron that had accumulated over the years because of blood transfusions."
The paper, titled "Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassemia," is available in the online publication of Nature at www.nature.com.
"We believe the human findings in beta-thalassemia, as well as the recently published data in Science on two patients with childhood cerebral adrenoleukodystrophy (CCALD), highlight the significant opportunity for bluebird bio's gene therapy platform to help patients with severe genetic disorders," said Nick Leschly, president and CEO of bluebird bio. "We are committed to building a world-class company in gene therapy led by outstanding people as we move aggressively forward with multiple clinical studies, including our ongoing clinical trials for the development of LentiGlobin for beta-thalassemia and our product for Page: 1 2 3 Related biology technology :1
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