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Pre-clinical development of a lentiviral vector expressing the anti-sickling beta AS3 globin for gene therapy for sickle-cell disease

  • Author(s): Poletti, V;
  • Charrier, S;
  • Urbinati, F;
  • Hollis, RP;
  • Corre, G;
  • Martin, S;
  • Weber, L;
  • Rothe, M;
  • Schambach, A;
  • Miccio, A;
  • Cavazzana, M;
  • Kohn, DB;
  • Mavilio, F
  • et al.
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