Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model

• Robert A. French,
• Benjamin J. Samelson-Jones,
• Glenn P. Niemeyer,
• Clinton D. Lothrop, Jr,
• Elizabeth P. Merricks,
• Timothy C. Nichols,
• Valder R. Arruda

Affiliations

Robert A. French

Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA;

Benjamin J. Samelson-Jones

Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA;

Glenn P. Niemeyer

Department of Biochemistry, University of Alabama at Birmingham, Birmingham, AL;

Clinton D. Lothrop, Jr

Department of Biochemistry, University of Alabama at Birmingham, Birmingham, AL;

Elizabeth P. Merricks

Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC;

Timothy C. Nichols

Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC;

Valder R. Arruda

Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA;; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA;; Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA; Valder R. Arruda, Children's Hospital of Philadelphia, 3501 Civic Center Blvd, 5056 Colket Translational Research Center, Philadelphia, PA 19104;