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    • AAV5-Factor VIII Gene Transfer in Severe Hemophilia A. 

      Rangarajan, S; Walsh, L; Lester, W; Perry, D; Madan, B; Laffan, M; Yu, H; Vettermann, C; Pierce, GF; Wong, WY (2017-12-28)
      BACKGROUND: Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, soft tissue, and the central nervous system. Although successful gene transfer has been reported in patients with hemophilia ...