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On va prélever des CSH à ces enfants, qui sont CD34+. Ensuite on les modifie .........pour leur amener le gène manquant avec un .........On va détruire les CSH malade du patient, avant de lui réinjecter ses cellules modifiées (via greffe de moëlle osseuse). La différenciation des ......... devient possible.Problèmes : les rétrovirus ont un risque oncogène, on a donc pour effet secndaire à cette thérapie des leucémies aiguë lymphoblastiques.

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