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

Author

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  • Marina Cavazzana-Calvo

    (Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Inserm/Assistance Publique–Hôpitaux de Paris, Paris 75015, France
    University Paris-Descartes)

  • Emmanuel Payen

    (CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI)
    Inserm U962 and University Paris XI, CEA-iMETI, Fontenay-aux-Roses 92265, France
    Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France)

  • Olivier Negre

    (CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI)
    Inserm U962 and University Paris XI, CEA-iMETI, Fontenay-aux-Roses 92265, France
    Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France
    Genetix-France, CEA-iMETI, Fontenay-aux-Roses 92265, France)

  • Gary Wang

    (University of Pennsylvania School of Medicine)

  • Kathleen Hehir

    (Genetix Pharmaceuticals)

  • Floriane Fusil

    (CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI)
    Inserm U962 and University Paris XI, CEA-iMETI, Fontenay-aux-Roses 92265, France
    Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France)

  • Julian Down

    (Genetix Pharmaceuticals)

  • Maria Denaro

    (Genetix Pharmaceuticals)

  • Troy Brady

    (University of Pennsylvania School of Medicine)

  • Karen Westerman

    (Genetix Pharmaceuticals
    Brigham & Women’s Hospital and Harvard Medical School)

  • 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
    University Paris-Descartes)

  • Riccardo Sgarra

    (University of Trieste)

  • Leila Maouche-Chrétien

    (CEA, Institute of Emerging Diseases and Innovative Therapies (iMETI)
    Inserm U962 and University Paris XI, CEA-iMETI, Fontenay-aux-Roses 92265, France)

  • 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)
    Inserm U962 and University Paris XI, CEA-iMETI, Fontenay-aux-Roses 92265, France
    Brigham & Women’s Hospital and Harvard Medical School)

  • 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
    University Paris-Descartes)

  • 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)
    Inserm U962 and University Paris XI, CEA-iMETI, Fontenay-aux-Roses 92265, France
    Bone Marrow Transplantation and Biochemistry, University Paris VII, Institute of Hematology, Hôpital Saint-Louis, AP-HP, Paris 75010, France)

  • 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
    University Paris-Descartes)

  • Philippe Leboulch

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.

Suggested Citation

  • 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-Leg, 2010. "Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia," Nature, Nature, vol. 467(7313), pages 318-322, September.
    • Handle: RePEc:nat:nature:v:467:y:2010:i:7313:d:10.1038_nature09328
    DOI: 10.1038/nature09328
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    Cited by:

    1. Suk See Ravin & Siyuan Liu & Colin L. Sweeney & Julie Brault & Narda Whiting-Theobald & Michelle Ma & Taylor Liu & Uimook Choi & Janet Lee & Sandra Anaya O’Brien & Priscilla Quackenbush & Tyra Estwick, 2022. "Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency," Nature Communications, Nature, vol. 13(1), pages 1-15, December.
    2. Sebastian Wagner & Christoph Baldow & Andrea Calabria & Laura Rudilosso & Pierangela Gallina & Eugenio Montini & Daniela Cesana & Ingmar Glauche, 2022. "Clonal reconstruction from co-occurrence of vector integration sites accurately quantifies expanding clones in vivo," Nature Communications, Nature, vol. 13(1), pages 1-12, December.
    3. Arianna Moiani & Gil Letort & Sabrina Lizot & Anne Chalumeau & Chloe Foray & Tristan Felix & Diane Clerre & Sonal Temburni-Blake & Patrick Hong & Sophie Leduc & Noemie Pinard & Alan Marechal & Eduardo, 2024. "Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells," Nature Communications, Nature, vol. 15(1), pages 1-21, December.
    4. Francesca Tucci & Stefania Galimberti & Luigi Naldini & Maria Grazia Valsecchi & Alessandro Aiuti, 2022. "A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders," Nature Communications, Nature, vol. 13(1), pages 1-13, December.

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