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

Cavazzana-Calvo, Marina; Payen, Emmanuel; Negre, Olivier; Wang, Gary; Hehir, Kathleen; Fusil, Floriane; Down, Julian; Denaro, Maria; Brady, Troy; Westerman, Karen; Cavallesco, Resy; Gillet-Legrand, Beatrix; Caccavelli, Laure; Sgarra, Riccardo; Maouche-Chrétien, Leila; Bernaudin, Françoise; Girot, Robert; Dorazio, Ronald; Mulder, Geert-Jan; Polack, Axel; Bank, Arthur; Soulier, Jean; Larghero, Jérôme; Kabbara, Nabil; Dalle, Bruno; Gourmel, Bernard; Socie, Gérard; Chrétien, Stany; Cartier, Nathalie; Aubourg, Patrick; Fischer, Alain; Cornetta, Kenneth; Galacteros, Frédéric; Beuzard, Yves; Gluckman, Eliane; Bushman, Frederick; Hacein-Bey-Abina, Salima; Leboulch, Philippe

Cavazzana-Calvo, Marina; Payen, Emmanuel; Negre, Olivier; Wang, Gary; Hehir, Kathleen; Fusil, Floriane; Down, Julian; Denaro, Maria; Brady, Troy; Westerman, Karen; Cavallesco, Resy; Gillet-Legrand, Beatrix; Caccavelli, Laure; Sgarra, Riccardo; Maouche-Chrétien, Leila; Bernaudin, Françoise; Girot, Robert; Dorazio, Ronald; Mulder, Geert-Jan; Polack, Axel; Bank, Arthur; Soulier, Jean; Larghero, Jérôme; Kabbara, Nabil; Dalle, Bruno; Gourmel, Bernard; Socie, Gérard; Chrétien, Stany; Cartier, Nathalie; Aubourg, Patrick; Fischer, Alain; Cornetta, Kenneth; Galacteros, Frédéric; Beuzard, Yves; Gluckman, Eliane; Bushman, Frederick; Hacein-Bey-Abina, Salima; Leboulch, Philippe.

Affiliation

Cavazzana-Calvo M; Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Inserm/Assistance Publique-Hôpitaux de Paris, Paris 75015, France.

Nature ; 467(7313): 318-22, 2010 Sep 16.

Article in En | MEDLINE | ID: mdl-20844535

Subject(s)

Blood Transfusion; Genetic Therapy; HMGA2 Protein/metabolism; beta-Globins/genetics; beta-Globins/metabolism; beta-Thalassemia/genetics; beta-Thalassemia/therapy; Adolescent; Blood Cells/cytology; Blood Cells/metabolism; Bone Marrow Cells/cytology; Bone Marrow Cells/metabolism; Child, Preschool; Clone Cells/metabolism; Gene Expression; Genetic Vectors/genetics; HMGA2 Protein/genetics; Homeostasis; Humans; Lentivirus/genetics; Male; MicroRNAs/genetics; Organ Specificity; RNA, Messenger/analysis; RNA, Messenger/genetics; Time Factors; Transcriptional Activation; Young Adult; beta-Thalassemia/metabolism

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Blood Transfusion / Genetic Therapy / Beta-Thalassemia / HMGA2 Protein / Beta-Globins Limits: Adolescent / Adult / Child, preschool / Humans / Male Language: En Journal: Nature Year: 2010 Type: Article Affiliation country: France

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