A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A.
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L. Naldini | M. Ozelo | B. Brown | P. McCray | D. Lillicrap | C. Hough | A. Tuttle | Hideto Matsui | A. Labelle | C. Hegadorn | Brian D. Brown | E. Burnett | Erin Burnett