Katherine A High, M.D.


Katherine A. High, M.D.

CCMT Director 3501 Civic Center Blvd.
Department of Pediatrics Colket Room 5060 Division Hematology Philadelphia, PA 19104   (215) 590-4521   This email address is being protected from spambots. You need JavaScript enabled to view it.


1. High KA, Lee HB, and Turner DT. Autoacceleration of free radical polymerization. 4. Predissolved polymer. Macromolecules 12:332-337, 1979

2. Weinstein P, High KA, D’Ercole AJ, and Jennette JC. Insulin resistance due to receptor antibodies: A complication of progressive systemic sclerosis. Arthrit Rheumat 23:101-105, 1980.

3. Lambert SR, High KA, Cotlier C, and Benz EJ, Jr. Reversible serous retinal detachments in two patients with thrombotic thrombocytopenic purpura. Arch Ophthalmol 103:1172-1174, 1985.

4. High KA, Stolle C, Schneider JW, Hu W, and Benz EJ, Jr. C-myc gene inactivation during induced maturation of HL-60 cells: Transcriptional repression and loss of a specific DNAse I hypersensitive site. J Clin Invest 79:93-99, 1987.

5. Macik BG, Gabriel DA, White GC, High KA, and Roberts HR. The use of high dose intravenous gammaglobulin in acquired von Willebrand’s syndrome. Arch Pathol Lab Med 112:143-146, 1988.

6. Huang MN, Kasper C, Roberts HR, Stafford DW, and High KA. Molecular defect in F.IXHilo, a hemophilia Bm variant: Arg→Gln at the carboxyterminal cleavage site of the activation peptide. Blood 73:718-721, 1989.

7. Monroe DM, McCord DM, Huang MN, High KA, Lundblad RL, Kasper CK, and Roberts HR. Functional consequences of an Arg180 to Gln mutation in Factor IX Hilo. Blood 73:1540-1544, 1989.

8. Evans JP, Watzke HH, Ware JL, Stafford DW, and High KA. Molecular cloning of a cDNA encoding canine F.IX. Blood 74:207-212, 1989.

9. Dent GA, Ayscue LH, High KA, L’Eglise MC, and Ross DW. Flow cytometric correlation of the c-myc oncoprotein and cell cycle kinetics of HL60 leukaemia during induced maturation with cytosine arabinoside and dimethylsulphoxide. Cell and Tissue Kinetics 22:1-11, 1989.

10. Reddy SV, Zhou ZQ, Rao KJ, Scott JP, Watzke H, High KA, and Jagadeeswaran P. Molecular characterization of human Factor X San Antonio. Blood 74:1486-1490, 1989.

11. Evans JP, Brinkhous KM, Brayer GD, Reisner HM, and High KA. Canine hemophilia B resulting from a point mutation with unusual consequences. Proc Natl Acad Sci USA 86:10095-10099, 1989.

12. Lozier JN, Monroe DM, Stanfield-Oakley SA, Lin SW, Smith KJ, Roberts HR, and High KA. Factor IXNew London. Substitution of proline for glutamine at position 50 causes severe hemophilia B. Blood 75:1097-1104, 1990.

13. Watzke HH, Lechner K, Roberts HR, Reddy SV, Welsch DJ, Friedman P, Mahr G, Jagadeeswaran P, Monroe DM, and High KA. Molecular defect (Gla+14→Lys) and its functional consequences in a hereditary factor X deficiency (Factor X “Vorarlberg”). J Biol Chem 265:11982-11989, 1990.

14. Giannelli F, Green PM, High KA, Lozier JN, Lillicrap DP, Ludwig M, Olek K, Reitsma PH, Goossens M, Yoshioka A, Sommer S, Paxman B, and Brownlee GG. Haemophilia B:- database of point mutations and short additions and deletions. Nucleic Acids Res 18:4053-4059, 1990; Nucleic Acids Res 19:2193-2219, 1991; Nucleic Acids Res 20:2027-2063, 1992; Nucleic Acids Res 21:3075-3087, 1993.

15. Huang MN and High KA. Efficient subcloning of DNA fragments amplified by crude oligonucleotides. Biotechniques 9(6):710-711, 1990.

16. Wallmark A, Rose VL, Ho C, and High KA. A Nla IV polymorphism within the human factor X gene. Nucleic Acids Res 19:4022, 1991.

17. Watzke HH, Wallmark A, Hamaguchi N, Giardina P, Stafford DW, and High KA. Factor XSanto Domingo. Evidence that the severe clinical phenotype arises from a mutation blocking secretion. J Clin Invest 88:1685-1689, 1991.

18. Huang MN, Hung HL, Stanfield-Oakley SA, and High KA. Characterization of the human blood coagulation factor X promoter. J Biol Chem 267:15440-15446, 1992.

19. Stern RA, van der Horst CM, Hooper SR, Bloodgood KM, and High KA. Zidovudine overdose in an asymptomatic HIV seropositive patient with hemophilia. Psychosomatics 4:454-457, 1992.

20. Racchi M, Watzke HH, High KA, and Lively MO. Human coagulation factor X deficiency caused by a mutant signal peptide that blocks cleavage by signal peptidase but not targeting and translocation to the endoplasmic reticulum. J Biol Chem 268(8):5735-5740, 1993.

21. Rose VL, Dermott SC, Murray BF, McIver MM, High KA, and Oberhardt BJ. Decentralized testing for PT and activated PTT using a dry chemistry portable analyzer. Arch Pathol Lab Med 117(6):611-617, 1993.

22. Sridhara S, Clarke BJ, Ofosu FA, High KA, and Blajchman MA. The direct binding of human factor VII in plasma to recombinant human tissue factor. Thrombosis Research 70:307-316, 1993.

23. Chaing S, Clarke B, Sridhara S, Chu K, Friedman P, VanDusen W, Roberts HR, Blajchman M, Monroe DM, and High KA. Severe Factor VII deficiency due to mutations abolishing the cleavage site for activation and altering binding to tissue factor. Blood 83:3524-3535, 1994.

24. Lozier JN, Thompson AR, Hu PC, Read M, Brinkhous KM, High KA, and Curiel DT. Efficient transfection of primary cells in a canine hemophilia B model using adenovirus-polylysine-DNA complexes. Human Gene Therapy 5:313-322, 1994.

25. Chaing SH, Wallmark A, Berntorp E, and High KA. A NlaIII polymorphism within the human factor VII gene. Human Genetics 93(6):722-723, 1994.

26. Bi L, Lawler AM, Antonarakis SE, High KA, Gearhart JD and Kazazian HH, Jr. Targeted disruption of the mouse factor VIII gene produces a model for haemophilia A. Nature Genetics 10: 119-121, 1995.

27. Pollak ES, Hung HL, Godin W, Overton GC and High KA. Functional characterization of the human Factor VII 5’-flanking region. J Biol Chem 271:1738-1747, 1996.

28. Hung HL and High KA. Liver-enriched transcription factor HNF-4 and ubiquitous factor NF-Y are critical for expression of blood coagulation factor X. J Biol Chem 271:2323-2331, 1996.

29. Larson P, Stanfield-Oakley SA, VanDusen WJ, Kasper CK, Smith KJ, Monroe, DM, High KA. Structural integrity of the γ-carboxyglutamic acid domain of human blood coagulation factor IXa is required for its binding to cofactor VIIIa. J Biol Chem 271:3869-3875, 1996.

30. Walter J, You Q, Hagstrom JN, Sands M, High KA. Successful expression of human factor IX following repeat administration of an adenoviral vector in mice. Proc Natl Acad Sci USA 93:3056-3061, 1996.

31. Chu K, Wu S, Stanley T, Stafford DW and High KA. A mutation in the propeptide of Factor IX leads to warfarin sensitivity by a novel mechanism. J Clin Invest 98:1619-1625, 1996.

32. Sridhara S, Chaing S, High KA, Blajchman MA and Clarke BJ. Activation of a recombinant human factor VII structural analogue alters its affinity of binding to tissue factor. American J. Hematology 53:66-71, 1996.

33. Arbini AA, Pollak ES, Bayleran JK, High KA and Bauer KA. Severe factor VII deficiency due to a mutation disrupting a hepatocyte nuclear factor 4 binding site in the factor VII promoter. Blood 89:176-182, 1997.

34. Fisher KJ, Jooss K, Alston J, Yang Y, Haecker SE, High KA, Pathak R, Raper SE, and Wilson JM. Recombinant adeno-associated virus for muscle directed gene therapy. Nature Medicine 3:306-312, 1997.

35. Wu SM, Stafford DW, Frazier LD, Fu YY, High KA, Chu K, Sanchez-Vega B, Solera J. Genomic sequence and transcription start site for the human γ-glutamyl carboxylase. Blood 89:4058-4062, 1997.

36. Ambrosini G, Plescia J, Chu KC, High KA and Altieri DC. Activation-dependent exposure of the inter-EGF sequence Leu83-Leu88 in factor Xa mediates ligand binding to effector cell protease receptor-1 (EPR-1). J Biol Chem 272:8340-8345, 1997.

37. Herzog RW, Hagstrom JN, Kung SH, Tai SJ, Wilson JM, Fisher KJ, High KA. Stable gene transfer and expression of human blood coagulation F.IX after intramuscular injection of recombinant AAV. Proc Natl Acad Sci USA 94: 5804-5809, 1997.

38. Kung SH, Hagstrom JN, Cass D, Tai SJ, Lin HF, Stafford DW, High KA. Human F.IX corrects the bleeding diathesis of mice with hemophilia B. Blood 91:784-790, 1998.

39. Larson PJ, Camire R, Wong D, Fasano NC, Monroe DM, Tracy PB, High KA. Structure/function analyses of recombinant variants of human factor Xa: Factor Xa incorporation into prothrombinase on the thrombin-activated platelet surface is not mimicked by synthetic phospholipid vesicles. Biochemistry 37:5029-5038, 1998.

40. Nakai H, Herzog RW, Hagstrom JN, Walter J, Kung SH, Yang EY, Tai SJ, Iwaki Y, Kurtzman GJ, Fisher KJ, colossi P, Couto LB, High KA. AAV-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood 91:4600-4607, 1998.

41. Carew JA, Pollak ES, High KA, Bauer KA. Severe factor VII deficiency due to a mutation disrupting an Sp1 binding site in the F.VII promoter. Blood 92:1639-1645, 1998.

42. Hagstrom JN, Walter J, Bluebond-Langner R, Amatniek JC, Manno CS, High KA. Prevalence of the factor V leiden mutation in children and neonates with thromboembolic disease. J. Peds. 133:777-781, 1998.

43. Herzog RW, Yang EY, Couto LB, Hagstrom JN, Elwell D, Fields PA, Burton M, Bellinger D, Read MS, Brinkhous KM, Podsakoff GM, Nichols TC, Kurtzman GJ, High KA. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nature Medicine 5:56-63, 1999.

44. Stanley TB, Humphries J, High KA, Stafford DW. Amino acids responsible for reduced affinities of vitamin K-dependent propeptides for the carboxylase. Biochemistry 38:15681-15687, 1999.

45. Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland, A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C., Skarsgard E, Flake AW and High KA. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nature Genetics 24:257-261, 2000.

46. Hagstrom JN, Couto, LB, Scallan C, Burton M, McCleland ML, Fields PA, Arruda VR, Herzog RW, High KA. Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter. Blood 95:2536-2542, 2000.

47. Fields PA, Kowalczyk DW, Arruda VR, Armstrong E, McCleland ML, Hagstrom JN, Pasi KJ, Ertl HCJ, Herzog RW, and High KA. Role of vector in activation of T cell subsets in immune responses against the secreted transgene product F.IX. Molecular Therapy, 1:225-235, 2000.

48. Herzog RW, Arruda VR, Fisher TH, Read MS, Nichols TC, and High KA. Absence of circulating factor IX antigen in hemophilia B dogs of the UNC-Chapel Hill colony. Thrombosis and Haemostasis 84:352-354, 2000.

49. Camire, RM, Larson PJ, Stafford DW, High KA. Enhanced γ-carboxylation of recombinant Factor X using a chimeric construct containing the prothrombin propeptide. Biochemistry, 39:14322-14329, 2000.

50. Arruda VR, Hagstrom JN, Deitch J, Heiman-Patterson T, Camire R, Chu K, Fields PA, Herzog RW, Couto LB, Larson PJ, and High KA. Posttranslational modifications of recombinant myotube-synthesized human factor IX. Blood 97:130-138, 2001.

51. Hung, H.L. Pollak ES, Kudaravalli RD, Chu K and High K.A. Regulation of human coagulation factor X gene expression by GATA-4 and the Sp family of transcription factors. Blood 97:946-951, 2001.

52. Fields P, Armstrong E, Hagstrom JN, Arruda VR, Murphy ML, Farrell JP, High KA, Herzog RW. Intravenous administration of an E1,E3-deleted adenoviral vector induces tolerance to a Factor IX in C57BL/6 mice. Gene Therapy 8:354-361, 2001.

53. Herzog RW, Mount MJ, Arruda VR, High KA, Lothrop CD, Jr. Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation. Molecular Therapy 4:192-200, 2001.

54. Fields PA, Arruda VR, Armstrong E, Chu K, Mingozzi F, Hagstrom JN, Herzog RW, High, KA. Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of factor IX. Molecular Therapy 4:201-210, 2001.

55. Arruda, VR, Fields PA, Milner R, Wainwright L, DeMiguel MP, Donovan PJ, Herzog RW, Nichols TC, Biegel J, Razavi M, Dake M, Huff D, Flake AW, Couto L, Kay MA, High KA. Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males. Molecular Therapy 4:586-592, 2001.

56. Mount MJ, Herzog RW, Tillson M, Goodman SA, Robinson N, McCleland ML, Bellinger D, Nichols TC, Arruda VR, Lothrop Jr. CD, High KA. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood 99:2670-2676, 2002. See commentary Blood 99:2635.

57. Toso R, Pinotti M, High KA, Pollak ES, Bernardi F. A frequent human coagulation Factor VII mutation (A294V, c152) in loop 140s affects the interaction with activators, tissue factor and substrates. Biochem J 363:411-416, 2002.

58. Herzog RW, Fields PA, Arruda VR, Brubaker JO, Armstrong E, McClintock D, Bellinger DA, Couto LB, Nichols TC, High KA. Influence of vector dose on Factor IX-specific T and B cell responses in muscle-directed gene therapy for hemophilia B. Human Gene Therapy 13:1281-1291, 2002.

59. Mingozzi F, Schüttrumpf J, Arruda VR, Liu Y, Liu Y-L, High KA, Xiao W, Herzog RW. Improved hepatic gene transfer using an adeno-associated virus serotype-5 vector. J. Virology 76:10497-10502, 2002.

60. Toso R, Bernardi F, Tidd T, Pinotti M, Camire RM, Marchetti G, High KA, Pollak ES. Factor VII mutant V154G models a zymogen-like form of Factor VIIa. Biochem J 369:563-571, 2003.

61. Manno CS, Chew A, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DGB, Johnson FA, McClelland A, Scallan C, Skarsgaard, E, Flake AW, Kay MA, High KA, Glader B. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101:2963-2972, 2003. [Epub ahead of print 2002 Dec. 19].

62. High KA, Herzog R, Arruda V. AAV-mediated gene transfer to liver. Blood 101:3338-3339, 2003 (letter).

63. Gericitano J, Matthias C, Mick, R. Duffy KM, Luger S, Stadtmauer EA, Schuster SJ, Tsai D, Nasta S, Berlin J, Phillips DK, High KA, Porter DL. Homocysteine and prothrombin fragment F 1+2 levels in patients with veno-occlusive disease after stem cell transplantation. Journal of Hematotherapy and Stem Cell Research 12:215-223, 2003.

64. Mingozzi F, Liu Y-L, Dobrzynski E, Kaufhold A, Liu JH, Wang YQ, Arruda VR, High KA, Herzog RW. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. Journal of Clinical Investigation 111:1347-56, 2003.

65. Kohn DB, Sadelain M, Dunbar C, Bodine D, Kiem H-P, Candotti F, Tisdale J, Riviere I, Blau CA, Richard RE, Sorrentino B, Nolta J, Malech, Brenner M, Cornetta K, Cavagnaro J, High K, Glorioso J. American Society of Gene Therapy (ASGT) Ad Hoc Subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther 8:180-187, 2003.

66. Arruda VR, Schuettrumpf J, Herzog RW, Nichols TC, Robinson N, Lofti Y, Mingozzi F, Xiao W, Couto LB, High KA. Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1. Blood 103:85-92, 2004. [Epub 2003 Sep 11].

67. Margaritis P, Arruda VR, Aljamali, M, Camire R, Schlachterman A, High KA. Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated FVII. J Clinical Investigation 113:1025-1031, 2004.

68. Sabatino D, Armstrong AE, Edmondson S, Pleimes M, Schuettrumpf, J, Fitzgerald J, Herzog RW, Arruda VR, High KA. Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene. Blood 104:2767-2774, 2004. [Epub 2004 Jun 24].

69. Whinna HC, Lesesky EB, Monroe DM, High KA, Larson PJ, Church FC. Role of the gamma-carboxyglutamic acid domain of activated factor X in the presence of calcium during inhibition by antithrombin-heparin. J Thromb Haemost 2:1127-1134, 2004.

70. Liu YL, Mingozzi F, Rodriguez-Colon SM, Dobrzynski E, Suzuki T, High KA, Herzog RW. Therapeutic Levels of Factor IX Expression Using a Muscle-Specific Promoter and Adeno-Associated Virus Serotype 1 Vector. Hum Gene Ther 15:783-792, 2004.

71. Hauck B, Zhao W, High K, Xiao W. Intracellular Viral Processing, Not Single-Stranded DNA Accumulation, Is Crucial for Recombinant Adeno-Associated Virus Transduction. J Virol 78:13678-13686, 2004.

72. Arruda VR, Stedman HH, Nichols TC, Haskins ME, Nicholson M, Herzog RW, Couto LB, High KA. Regional intravascular infusion of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood 105:3458-3464, 2005. [Epub ahead of print 2004 Oct. 12].

73. Zhang H-G, High KA, Wu Q, Pang PA, Schlachterman A, Yu S, Hsu H-C, Mountz JD. Genetic analysis of the antibody response to AAV2 and Factor IX, Mol. Ther 11:866-874, 2005.

74. Sabatino DE, Mingozzi F, Hui DJ, Chen H, Colosi P, Ertl HCJ, High KA. Identification of mouse AAV capsid-specific CD8+ T cell epitopes. Mol Ther 12:1023-1033, 2005, [Epub ahead of print 2005 Nov. 2].

75. Manno CS, Arruda VR, Pierce GF, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt, P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA*, Kay MA. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nature Med 12:342-347, 2006. *Corresponding author.

76. Cao O, Armstrong E, Schlachterman A, Wang L, Okita DK, Conti-Fine B, High KA, Herzog RW. Immune deviation by musosal antigen administration suppresses gene transfer-induced inhibitor formation to factor IX. Blood 108:480-486, 2006 [Epub 2006 Mar 16].

77. Jiang H, Pierce GF, Ozelo MC, dePaula EV, Vargas JA, Smith P, Sommer J, Luk A, Manno CS, High KA, Arruda VR. Evidence of multi-year factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol Ther 14:452-455, 2006 [Epub 2006 Jul 5].

78. Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy, D, Vargas JA, Zhou S, Scallan CD, Sommer J, Vijay S, Warren D, Mingozzi F, High KA, Pierce GF. Effects of transient immuno-suppression on adeno associated virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 108:3321-3328, 2006 [Epub ahead of print July 25].

79. Callan MB, Aljamali MN, Margaritis P, Griot-Wenk ME, Pollak ES, Werner P, Giger U, High KA. A novel missense mutation responsible for factor VII deficiency in research Beagle colonies. J Thromb Haemost 4:2616-2622, 2006 [Epub 2006 Sep 8].

80. Qu G, Bahr-Davidson J, Prado J, Tai A, Cataniag F, McDonnell J, Zhou J, Hauck B, Luna J, Sommer JM, Smith P, Zhou S, Colosi P, High KA, Pierce GF, Wright JF. Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography. J Virol Meth 104:183-192, 2007. [Epub 2006 Dec 27].

81. Li H, Murphy S, Giles-Davis W, Edmonson S, Xiang Z, Li Y, Lasaro MO, High KA, Ertl HCJ. Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes, Mol Ther 15:792-800, 2007 [Epub 2007 Jan 23].

82. Mingozzi F*, Maus MV*, Hui DJ*, Sabatino DE, Murphy SL, Rasko JE, Ragni MV, Manno CS, Sommer JS, Jiang H, Pierce GF, Ertl HC, High KA. CD8+ T cell responses to adeno-associated virus capsid in humans. *Authors contributed equally to ms. Nature Medicine13:419-422, 2007. [Epub 2007 March 18].

83. Ingerslev J, Herlin T, Sorensen B, Clausen N, Chu KC, High KA. Severe factor X deficiency in a pair of siblings: clinical presentation, phenotypic and genotypic features, prenatal diagnosis and treatment. Haemophilia 13:334-336, 2007.

84. Sabatino DE, MacKenzie TC, Campagnoli C, Liu Y-L, Flake AW, High KA. Persistent expression of human F.IX after tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice. Molecular Therapy 15:1677-1685, 2007 [Epub 2007 June 12].

85. Mingozzi F, Hasbrouck NC, Basner-Tschakarjan E, Edmonson SA, Hui DJ, Sabatino DE, Zhou S, Wright JF, Jiang H, Pierce GF, Arruda VR, High KA. Modulation of tolerance to the transgene product in a non-human primate model of AAV-mediated gene transfer to liver. Blood 110:2334-2341, 2007 [Epub 2007 Jul 3].

86. Yang C, Feng J, Song W, Wang J, Tsai B, Zhang Y, Hill KA, Margaritis P, High KA, Sommer SS. A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin. Proc Natl Acad Sci USA 104:15394-15399, 2007 [Epub 2007 Sep 19].

87. Murphy SL, Li H, Zhou S, Schlachterman A, High KA. Prolonged susceptibility to antibody-mediated neutralization for adeno-associated vectors targeted to the liver. Mol Ther 16:138-145, 2008 [Epub 2007 Oct 23].

88. Tai SJ, Herzog RW, Arruda VR, Chu K, Golden JA, Labosky PA, High KA. A viable mouse model of factor X deficiency provides evidence for maternal transfer of factor X. Journal Thrombosis Haemostasis 6:339-345, 2008 [Epub 2007 Nov 23].

89. Bennicelli J, Wright JF, Jacobs J, Komaromy A, Hauck B, Zelenaia O, Mingozzi F, Hui D, Chung D, Rex TS, Wei Z, Zeiss C, Arruda VR, Puch EN, Acland GM, Dell’Osso LF, High KA, Maguire AM, Bennett J. Reversal of blindness in animal models of Leber Congenital Amaurosis using optimized AAV2-mediated gene transfer. Molecular Therapy 16:458-465, 2008 [Epub 2008 Jan 22].

90. Aljamali MN, Margaritis P, Schlachterman A, Tai SJ, Roy E, Bunte R, Camire R, High KA. Long-term expression of murine activated factor VIIa is safe, but elevated levels cause premature mortality. J Clin Invest 118:1825-1834, 2008.

91. Toromanoff A, Chérel Y, Guilbaud M, Penaud-Budloo M, Snyder RO, Haskins ME, Deschamps J-Y, Guigand L, Podevin G, Arruda VR, High KA, Stedman HH, Rolling F, Anegon I, Moullier P, Le Guiner C. Safety and efficacy of regional intravenous (RI) versus intramuscular (IM) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle. Molecular Therapy 16:1291-1299, 2008 [Epub 2008 May 6].

92. Maguire AM*, Simonelli F*, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell’osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A*, High KA*, Bennett J*. Safety and efficacy of gene transfer for Leber’s Congenital Amaurosis. New England Journal of Medicine 358:2240-2248, 2008 [Epub 2008 April 28]. *Co-senior authors

93. Murphy SL, Bhagwat A, Edmonson S, Zhou S, High KA. High throughput screening and biophysical interrogation of hepatotrophic AAV. Mol Ther 16:1960-1967, 2008.

94. Stroes ESG, Nierman MC, Meulenberg J, Frannsen R, Twisk J, Henny P, Maas M, Zwinderman K, Ross C, Aronica E, High KA, Levi M, Kastelein JP, Kuivenhoven JA. Intramuscular administration of AAV1-lipoprotein Lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arterioscler Thromb Vasc Biol 28:2303-2304, 2008.

95. Hauck B, Murphy S, Smith PH, Qu G, Liu X, Zelenaia O, Mingozzi F, Sommer JM, High KA, Wright JF. Undetectable transcription of cap in a clinical AAV vector: Implications for pre-formed capsid in immune responses. Mol Ther 17:144-152, 2009.

96. Gupta S, Kühnich J, Mustafa A, Lhotak S, Schlachterman A, Slifker MJ, Klein-Szanto A, High KA, Austin RC, Kruger WD. Mouse models of cystathionine β-synthase deficiency reveal significant threshold effects of hyperhomocysteinemia. FASEB Journal 23:883-893, 2008.

97. Niemeyer G, Herzog RW, Mount JD, Arruda VR, Tillson DM, Hathcock JT, vanGinkel FW, High KA, Lothrop C. Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy. Blood 113:797-806, 2009

98. Murphy SL, Li H, Mingozzi F, Sabatino D, Hui D, Edmonson S, High KA. Diverse IgG subclass responses to adeno-associated virus infection and vector administration. J Med Virol 81:65-74, 2009.

99. Margaritis P, Roy E, Aljamali MN, Downey HD, Giger U, Zhou S, Merricks E., Dillow A, Nichols TC, High KA. Successful treatment of canine hemophilia by continuous expression of canine FVIIa. Blood 113:3682-3689, 2009.

100. Chen L, Lu H, Wang J, Sarkar R, Yang X, Wang H, High KA, Xiao W. Enhanced factor VIII heavy chain for gene therapy of hemophilia A. Mol Ther 17:417-424, 2009.

101. Favaro P, Downey HD, Zhou S, Wright JF, Hauck B, Mingozzi M, High KA, Arruda VA. Host and vector-dependent effects on the risk of germline transmission of AAV vectors. Mol Ther 17:1022-1030, 2009.

102. Pien GC, Basner-Tschakarjan E, Hui DJ, Mentlik A, Finn JD, Hasbrouck HC, Zhou S, Murphy SL, Maus MV, Mingozzi F, Orange JS, High KA. Capsid antigen presentation flags hepatocytes for destruction after transduction by adeno-associated viral vectors. J Clin Invest 119:1688-1695, 2009.

103. Li H, Lin S-W, Giles-Davis W, Li Y, Zhou D, Xiang ZQ, High KA, Ertl HCJ. A pre-clinical animal model to assess the effect of pre-existing immunity on AAV-mediated gene transfer. Mol Ther 17:1215-1224, 2009.

104. Mingozzi F, Meulenberg JJ, Hui DJ, Basner-Tschakarjan E, Hasbrouck NC, Edmonson SA, deJong A, Pos P, Twisk J, Hutnick NA, Betts MA, Kastelein JJ, Stroes ES, van Deventer SJ, High KA. AAV-1 mediated gene transfer to skeletal muscle in human results in dose-dependent activation of capsid-specific T cells. Blood 114:2077-2086, 2009.

105. Cao O, Hoffman BE, Moghimi B, Nayak S, Cooper M, Zhu S, Ertl HCJ, High KA, Herzog RW. Impact of the underlying mutation and the route of vector administration on immune response to factor IX in gene therapy for hemophilia B. Mol Ther 17:1733-1742, 2009

106. Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying G-s, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JIW, Hauck B, Zelenaia O, Zhu X, Raffini L, Coppieters F, DeBaere E, Shindler KS, Volpe NJ, Surace EM, Acerra C, Lyubarsky A, Redmond TM, Stone E, Sun J, Wellman McDonnell J, Leroy BP, Simonelli F, Bennett J. Age-dependent effects of RPE65 gene therapy for Leber’s congenital amaurosis: a phase 1 dose escalation trial. Lancet 374:1597-1605, 2009

107. Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli J, Rossi S, Marshall K, Banfi S, Surace EM, Sun J, Redmond TM, Zhu X, Shindler KS, Morgan J, Ying G-s, Acerra C, Maguire MG, Wright JF, Wellman McDonnell J, High KA, Bennett J, Auricchio A. Gene therapy for Leber’s congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther 18:643-650, 2010. [Epub 2009 Dec 1].

108 Finn JD, Hui D, Downey HD, Dunn D, Pien G, Mingozzi F, High KA. Proteasome inhibitors decrease AAV2 capsid-derived peptide epitope presentation on MHC Class I following transduction. Mol Ther 18:135,142, 2010. [Epub 2009 Nov 10]

109. Ayuso E, Mingozzi F, Montane J, Leon X, Anguela XM, Haurigot V, Edmonson S, Africa L, Zhou S, High KA, Boch F, Wright JF. High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency. Gene Therapy 17:503-10, 2010.

110. Amado D, Mingozzi F, Hui D, Bennicelli JL, Wei Z, Chan Y, Bote E, Grant RL, Golden JA, Narfstrom K, Syed NA, Orlin SE, Maguire AM, High KA, Bennett J. Safety and efficacy of subetinal readministration of a viral vector in large animals to treat congenital blindness. Sci Trans Med 2(21ra16):1-9, 2010, [Mar 3 Online issue].

111. Arruda VR, Stedman HH, Haurigot VA, Buchlis G, Baila S, Favaro P, Chen Y, Zhou S, Wright JF, Couto LB, Jiang H, Pierce GF, Bellinger DA, Mingozzi F, Nichols TC, High KA. Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. Blood 115:4678-4688, 2010 [Epub 2010 March 24].

112. Haurigot V, Mingozzi F, Buchlis G, Hui DJ, Chen Y, Arruda VR, Radu A, Franck HG, Wright J, 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 18:1318-1329, 2010, [Epub 2010 Apr 27].

113. Lin C-N, Kao C-Y, Miao CH, Hamaguchi N, Wu H-L, Shi G-Y, Liu Y-L, High KA, Lin S-W. Generation of a novel factor IX with augmented clotting activities in vitro and in vivo. J Thromb Haemost 8:1773-1783, 2010 [Epub 2010 May 21].

114. Li H, Malani N, Hamilton SR, Schlachterman A, Bussadori G, Edmonson SE, Shah R, Arruda VR, Mingozzi FM, Wright JF, Bushman FD, High KA. Assessing the potential for AAV vector genotoxicity in a murine model, Blood 117:3311-3319, 2011 [Epub 2010 Nov 24].

115. Favaro P, Finn JD, Siner J, Wright JF, High KA, Arruda VR. Safety of liver gene transfer following peripheral intravascular delivery of AAV-5 and AAV-6 in a large animal model. Hum Gene Ther March 8 [Epub ahead of print].

116. Li H, Tuyishime S, Wu TL, Giles-Davis W, Zhou D, Xiao W, High KA, Ertl HC. Adeno-associated virus vectors serotype 2 induced prolonged proliferation of capsid-specific CD8(+) T cells in mice. Mol Ther 19:536-546, 2011. [Epub 2010 Dec 14].

117. Nathwani AC, Rosales C, McIntosh J, Rastegariari G, Raj D, Nawathe S, Waddington S, Ng CYC, Zhou J, McCarville B, Bronson R, Jackson S, Allay J, Coleman J, Sleep S, Dunbar C, High KA, Mingozzi F, Valentine M, Gray JT, Nienhuis AW, Davidoff AM. Long term safety and efficacy following systemic administration of a self complementary adeno-associated viral vector encoding human FIX. Mol Ther 2011 Jan 18 [Epub ahead of print].

118. Zhou J, Yang X, Couto L, High KA, Qu G. PEG modulated column chromatography for purification of recombinant adeno-associated virus sero-type 9. J Virol Meth 173:99-107, 2011 Feb 3 [Epub 2011 Feb 3].

119. Margaritis P, Roy E, Faella A, Downey HD, Ivanciu L, Pavani G, Zhou S, Bunte RM, High KA. Catalytic domain modification and viral gene delivery of activated Factor VII confers hemostasis at reduced expression levels and vector doses in vivo. Blood 117:3974-3982, 2011 [Epub 2011 Feb 16].


Xavier Anguela (post-doc) This email address is being protected from spambots. You need JavaScript enabled to view it.

Etiena Basner-Tschakarjan (Research Associate) This email address is being protected from spambots. You need JavaScript enabled to view it.

George Buchlis (graduate student) This email address is being protected from spambots. You need JavaScript enabled to view it.

Yifeng Chen (Research Associate) This email address is being protected from spambots. You need JavaScript enabled to view it.

Robert Davidson (Research Associate) This email address is being protected from spambots. You need JavaScript enabled to view it.

Daniel Hui (post-doc) This email address is being protected from spambots. You need JavaScript enabled to view it.

Jianhua Liu (Research Assistant) This email address is being protected from spambots. You need JavaScript enabled to view it.

Katherine Marcucci (post-doc) This email address is being protected from spambots. You need JavaScript enabled to view it.

Rajiv Sharma (graduate student) This email address is being protected from spambots. You need JavaScript enabled to view it.

Mustafa Yazicioglu (post-doc) This email address is being protected from spambots. You need JavaScript enabled to view it.

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