Katherine A. High, MD Areas of Expertise: Hemophilia, Thrombosis, Molecular basis of blood coagulation, Gene therapy for hemophilia Locations: Main Campus Appointments and Referrals: 1-800-TRY-CHOP (1-800-879-2467) Education and Training Medical SchoolUniversity of North Carolina School of Medicine, Chapel Hill, NCResidencyNorth Carolina Memorial Hospital, Chapel Hill, NCFellowshipYale University School of Medicine, New Haven, Conn.Board CertificationAmerican Board of Internal MedicineAmerican Board of Internal Medicine, Hematology Titles and Academic Titles Investigator, Howard Hughes Medical InstituteAttending physician Departments and Services Division of Hematology Publications Papers Mingozzi F, Meulenberg JJ, Hui DJ, Basner-Tschakarjan E, Hasbrouck NC, Edmonson SA, Hutnick NA, Betts MR, Kastelein JJ, Stroes ES, High KA. AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells. Blood. 2009 Sep 3;114(10):2077-86. Epub 2009 Jun 8. Read abstract.Couto LB, High KA. Viral vector-mediated RNA interference. Curr Opin Pharmacol. 2010 Jul 8. [Epub ahead of print]. Read abstractHaurigot V, Mingozzi F, Buchlis G, Hui DJ, Chen Y, Basner-Tschakarjan E, Arruda VR, Radu A, Franck HG, Wright JF, Zhou S, Stedman HH, Bellinger DA, Nichols TC, High KA. Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs. Mol Ther. 2010 Jul;18(7):1318-29. Epub 2010 Apr 27. Read abstractMargaritis P, High KA. Gene therapy in haemophilia—going for cure?. Haemophilia. 2010 May;16 Suppl 3:24-8. Read abstract Pien GC, Basner-Tschakarjan E, Hui DJ, Mentlik AN, Finn JD, Hasbrouck NC, Zhou S, Murphy SL, Maus MV, Mingozzi F, Orange JS, High KA. Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J Clin Invest. 2009 Jun;119(6):1688-95. doi: 10.1172/JCI36891. Epub 2009 May 11. Read abstract. Finn JD, Hui D, Downey HD, Dunn D, Pien GC, Mingozzi F, Zhou S, High KA. Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction. Mol Ther. 2010 Jan;18(1):135-42. Epub 2009 Nov 10. Read abstract Patient Experience Ratings About the Patient Experience Rating System The Patient Experience Rating is an average of all responses to the care provider related questions shown below from our nationally-recognized Press Ganey Patient Satisfaction Survey. Patients that are treated in outpatient or hospital environments may receive different surveys, and the volume of responses will vary by question. Responses are measured on a scale of 1 to 5 with 5 being the best score. The comments are submitted by patients and families and reflect their views and opinions. The comments are not endorsed by and do not reflect the views of Children’s Hospital of Philadelphia.