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

Marina Cavazzana-Calvo, Emmanuel Payen, Olivier Negre, Gary Wang, Kathleen Hehir, Floriane Fusil, Julian Down, Maria Denaro, Troy Brady, Karen Westerman, Resy Cavallesco, Beatrix Gillet-Legrand, Laure Caccavelli, Riccardo Sgarra, Leila Maouche-Chrétien, Françoise Bernaudin, Robert Girot, Ronald Dorazio, Geert-Jan Mulder, Axel Polack, Arthur Bank, Jean Soulier, Jérôme Larghero, Nabil Kabbara, Bruno Dalle, Bernard Gourmel, Gérard Socie, Stany Chrétien, Nathalie Cartier, Patrick Aubourg, Alain Fischer, Kenneth Cornetta, Frédéric Galacteros, Yves Beuzard, Eliane Gluckman, Frederick Bushman, Salima Hacein-Bey-Abina and Philippe Leboulch ()
Additional contact information
Marina Cavazzana-Calvo: Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Inserm/Assistance Publique–Hôpitaux de Paris, Paris 75015, France
Emmanuel Payen: CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI)
Olivier Negre: CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI)
Gary Wang: University of Pennsylvania School of Medicine
Kathleen Hehir: Genetix Pharmaceuticals
Floriane Fusil: CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI)
Julian Down: Genetix Pharmaceuticals
Maria Denaro: Genetix Pharmaceuticals
Troy Brady: University of Pennsylvania School of Medicine
Karen Westerman: Genetix Pharmaceuticals
Resy Cavallesco: Brigham & Women’s Hospital and Harvard Medical School
Beatrix Gillet-Legrand: Genetix-France, CEA-iMETI, Fontenay-aux-Roses 92265, France
Laure Caccavelli: Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Inserm/Assistance Publique–Hôpitaux de Paris, Paris 75015, France
Riccardo Sgarra: University of Trieste
Leila Maouche-Chrétien: CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI)
Françoise Bernaudin: Centre Hospitalier Intercommunal de Créteil
Robert Girot: Hôpital Tenon, Paris 75020, France
Ronald Dorazio: Genetix Pharmaceuticals
Geert-Jan Mulder: Genetix Pharmaceuticals
Axel Polack: Genetix Pharmaceuticals
Arthur Bank: Columbia University College of Physicians and Surgeons
Jean Soulier: Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France
Jérôme Larghero: Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France
Nabil Kabbara: Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France
Bruno Dalle: Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France
Bernard Gourmel: Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France
Gérard Socie: Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France
Stany Chrétien: CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI)
Nathalie Cartier: Inserm UMR745, University Paris-Descartes
Patrick Aubourg: Inserm UMR745, University Paris-Descartes
Alain Fischer: Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Inserm/Assistance Publique–Hôpitaux de Paris, Paris 75015, France
Kenneth Cornetta: Indiana University
Frédéric Galacteros: Hopital Henri Mondor, AP-HP, Créteil 94000, France
Yves Beuzard: CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI)
Eliane Gluckman: Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France
Frederick Bushman: University of Pennsylvania School of Medicine
Salima Hacein-Bey-Abina: Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Inserm/Assistance Publique–Hôpitaux de Paris, Paris 75015, France

Nature, 2010, vol. 467, issue 7313, 318-322

Abstract: Gene therapy success Blood disorders caused by abnormal β-globin — β-thalassaemia and sickle cell disease — are the most prevalent inherited disorders worldwide, with patients often remaining dependent on blood transfusions throughout their lives. So a report of the successful use of gene therapy in a case of severe β-thalassaemia — using a lentiviral vector expressing the β-globin gene — is an eagerly awaited event. More than two years after gene transfer, the adult male patient has been transfusion-independent for 21 months. The therapeutic benefit seems to result from a dominant, myeloid-biased cell clone that may remain benign, although it could yet develop into leukaemia — a reminder that gene therapy is still at an early stage.

Date: 2010
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DOI: 10.1038/nature09328

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