Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.
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M. Ozelo | J. Sommer | K. High | C. Manno | V. Arruda | E. V. de Paula | G. Pierce | A. Luk | Haiyan Jiang | Joseph A. Vargas | Peter K. Smith | E. D. de Paula | P. Smith