Katherine A. High - Publications

Cell and Molecular Biology University of Pennsylvania, Philadelphia, PA, United States 
Immunology, Cell Biology, Molecular Biology

208 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2017 George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, ... ... High KA, et al. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. The New England Journal of Medicine. 377: 2215-2227. PMID 29211678 DOI: 10.1056/NEJMoa1708538  1
2017 Mingozzi F, High KA. Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape. Annual Review of Virology. 4: 511-534. PMID 28961410 DOI: 10.1146/annurev-virology-101416-041936  1
2017 Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, ... ... High KA, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet (London, England). PMID 28712537 DOI: 10.1016/S0140-6736(17)31868-8  0.36
2017 Ertl HC, High KA. The impact of AAV capsid-specific T cell responses on design and outcome of clinical gene transfer trials with recombinant AAV vectors - an evolving controversy. Human Gene Therapy. PMID 28042943 DOI: 10.1089/hum.2016.172  1
2016 Anguela XM, High KA. An edible switch for gene therapy. Nature Biotechnology. 34: 824-5. PMID 27504775 DOI: 10.1038/nbt.3645  1
2016 Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J, Elci OU, Chung DC, Sun J, Wright JF, Cross DR, Aravand P, Cyckowski LL, Bennicelli JL, Mingozzi F, ... ... High KA, et al. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Lancet (London, England). PMID 27375040 DOI: 10.1016/S0140-6736(16)30371-3  1
2016 Kumar SR, Markusic DM, Biswas M, High KA, Herzog RW. Clinical development of gene therapy: results and lessons from recent successes. Molecular Therapy. Methods & Clinical Development. 3: 16034. PMID 27257611 DOI: 10.1038/mtm.2016.34  1
2016 Vercauteren K, Hoffman BE, Zolotukhin I, Keeler GD, Xiao JW, Basner-Tschakarjan E, High KA, Ertl HC, Rice CM, Srivastava A, de Jong YP, Herzog RW. Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid. Molecular Therapy : the Journal of the American Society of Gene Therapy. 24: 1042-1049. PMID 27019999 DOI: 10.1038/mt.2016.61  1
2016 Callan MB, Haskins ME, Wang P, Zhou S, High KA, Arruda VR. Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs. Plos One. 11: e0151800. PMID 27011017 DOI: 10.1371/journal.pone.0151800  1
2015 Marcos-Contreras OA, Smith SM, Bellinger DA, Raymer RA, Merricks E, Faella A, Pavani G, Zhou S, Nichols TC, High KA, Margaritis P. Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII. Blood. PMID 26702064 DOI: 10.1182/blood-2015-09-671420  1
2015 Hui DJ, Edmonson SC, Podsakoff GM, Pien GC, Ivanciu L, Camire RM, Ertl H, Mingozzi F, High KA, Basner-Tschakarjan E. AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes. Molecular Therapy. Methods & Clinical Development. 2: 15029. PMID 26445723 DOI: 10.1038/mtm.2015.29  1
2015 Sharma R, Anguela XM, Doyon Y, Wechsler T, DeKelver RC, Sproul S, Paschon DE, Miller JC, Davidson RJ, Shivak D, Zhou S, Rieders J, Gregory PD, Holmes MC, Rebar EJ, ... High KA, et al. In vivo genome editing of the albumin locus as a platform for protein replacement therapy. Blood. PMID 26297739 DOI: 10.1182/blood-2014-12-615492  1
2015 Nichols TC, Whitford MH, Arruda VR, Stedman HH, Kay MA, High KA. Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs. Human Gene Therapy. Clinical Development. 26: 5-14. PMID 25675273 DOI: 10.1089/humc.2014.153  1
2015 Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, et al. Perspectives on best practices for gene therapy programs. Human Gene Therapy. 26: 127-33. PMID 25654329 DOI: 10.1089/hum.2014.147  1
2014 Nichols T, Whitford MH, Arruda VR, Stedman HH, Kay MA, High KA. Translational Data from AAV-Mediated Gene Therapy of Hemophilia B in Dogs. Human Gene Therapy. Clinical Development. PMID 25548971 DOI: 10.1089/hum.2014.153  1
2014 Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, ... ... High KA, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. The New England Journal of Medicine. 371: 1994-2004. PMID 25409372 DOI: 10.1056/NEJMoa1407309  1
2014 High KA. Gene therapy for hemophilia: the clot thickens. Human Gene Therapy. 25: 915-22. PMID 25397928 DOI: 10.1089/hum.2014.2541  1
2014 Wu TL, Li H, Faust SM, Chi E, Zhou S, Wright F, High KA, Ertl HC. CD8+ T cell recognition of epitopes within the capsid of adeno-associated virus 8-based gene transfer vectors depends on vectors' genome. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 42-51. PMID 24077034 DOI: 10.1038/mt.2013.218  1
2013 Markusic DM, Hoffman BE, Perrin GQ, Nayak S, Wang X, LoDuca PA, High KA, Herzog RW. Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies. Embo Molecular Medicine. 5: 1698-709. PMID 24106230 DOI: 10.1002/emmm.201302859  1
2013 Anguela XM, Sharma R, Doyon Y, Miller JC, Li H, Haurigot V, Rohde ME, Wong SY, Davidson RJ, Zhou S, Gregory PD, Holmes MC, High KA. Robust ZFN-mediated genome editing in adult hemophilic mice. Blood. 122: 3283-7. PMID 24085764 DOI: 10.1182/blood-2013-04-497354  1
2013 Yazicioglu MN, Monaldini L, Chu K, Khazi FR, Murphy SL, Huang H, Margaritis P, High KA. Cellular localization and characterization of cytosolic binding partners for Gla domain-containing proteins PRRG4 and PRRG2. The Journal of Biological Chemistry. 288: 25908-14. PMID 23873930 DOI: 10.1074/jbc.M113.484683  1
2013 Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, Yazicioglu M, Elkouby L, Hinderer CJ, Faella A, Howard C, Tai A, Podsakoff GM, Zhou S, Basner-Tschakarjan E, ... ... High KA, et al. Overcoming preexisting humoral immunity to AAV using capsid decoys. Science Translational Medicine. 5: 194ra92. PMID 23863832 DOI: 10.1126/scitranslmed.3005795  1
2013 Gil-Fariña I, Di Scala M, Vanrell L, Olagüe C, Vales A, High KA, Prieto J, Mingozzi F, Gonzalez-Aseguinolaza G. IL12-mediated liver inflammation reduces the formation of AAV transcriptionally active forms but has no effect over preexisting AAV transgene expression. Plos One. 8: e67748. PMID 23844082 DOI: 10.1371/journal.pone.0067748  1
2013 Buchlis G, Odorizzi P, Soto PC, Pearce OM, Hui DJ, Jordan MS, Varki A, Wherry EJ, High KA. Enhanced T cell function in a mouse model of human glycosylation. Journal of Immunology (Baltimore, Md. : 1950). 191: 228-37. PMID 23709682 DOI: 10.4049/jimmunol.1202905  1
2013 Mingozzi F, High KA. Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood. 122: 23-36. PMID 23596044 DOI: 10.1182/blood-2013-01-306647  1
2013 O'Reilly M, Kohn DB, Bartlett J, Benson J, Brooks PJ, Byrne BJ, Camozzi C, Cornetta K, Crystal RG, Fong Y, Gargiulo L, Gopal-Srivastava R, High KA, Jacobson SG, Jambou RC, et al. Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Human Gene Therapy. 24: 355-62. PMID 23517518 DOI: 10.1089/hum.2013.064  1
2013 Testa F, Maguire AM, Rossi S, Pierce EA, Melillo P, Marshall K, Banfi S, Surace EM, Sun J, Acerra C, Wright JF, Wellman J, High KA, Auricchio A, Bennett J, et al. Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Ophthalmology. 120: 1283-91. PMID 23474247 DOI: 10.1016/j.ophtha.2012.11.048  1
2013 Parzych EM, Li H, Yin X, Liu Q, Wu TL, Podsakoff GM, High KA, Levine MH, Ertl HC. Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans. Human Gene Therapy. 24: 431-42. PMID 23461589 DOI: 10.1089/hum.2012.246  1
2013 McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, ... ... High KA, et al. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood. 121: 3335-44. PMID 23426947 DOI: 10.1182/blood-2012-10-462200  1
2013 Callejas D, Mann CJ, Ayuso E, Lage R, Grifoll I, Roca C, Andaluz A, Ruiz-de Gopegui R, Montané J, Muñoz S, Ferre T, Haurigot V, Zhou S, Ruberte J, Mingozzi F, ... High KA, et al. Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy. Diabetes. 62: 1718-29. PMID 23378612 DOI: 10.2337/db12-1113  1
2013 Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, ... ... High KA, et al. Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 266-8. PMID 23369965 DOI: 10.1038/mt.2013.4  1
2013 Martino AT, Basner-Tschakarjan E, Markusic DM, Finn JD, Hinderer C, Zhou S, Ostrov DA, Srivastava A, Ertl HC, Terhorst C, High KA, Mingozzi F, Herzog RW. Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. Blood. 121: 2224-33. PMID 23325831 DOI: 10.1182/blood-2012-10-460733  1
2013 Mingozzi F, Chen Y, Edmonson SC, Zhou S, Thurlings RM, Tak PP, High KA, Vervoordeldonk MJ. Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue. Gene Therapy. 20: 417-24. PMID 22786533 DOI: 10.1038/gt.2012.55  1
2012 High KA. The gene therapy journey for hemophilia: are we there yet? Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program. 2012: 375-81. PMID 23233607 DOI: 10.1182/asheducation-2012.1.375  1
2012 Finn JD, Nichols TC, Svoronos N, Merricks EP, Bellenger DA, Zhou S, Simioni P, High KA, Arruda VR. The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy. Blood. 120: 4521-3. PMID 22919027 DOI: 10.1182/blood-2012-06-440123  1
2012 High KA. The gene therapy journey for hemophilia: are we there yet? Blood. 120: 4482-7. PMID 22829631 DOI: 10.1182/blood-2012-05-423210  1
2012 Hoffman BE, Ertl HC, Terhorst C, High KA, Herzog RW. Gene therapy research at the frontiers of viral immunology. Frontiers in Microbiology. 3: 182. PMID 22783235 DOI: 10.3389/fmicb.2012.00182  1
2012 Suhy DA, Kao SC, Mao T, Whiteley L, Denise H, Souberbielle B, Burdick AD, Hayes K, Wright JF, Lavender H, Roelvink P, Kolykhalov A, Brady K, Moschos SA, Hauck B, ... ... High KA, et al. Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 1737-49. PMID 22735378 DOI: 10.1038/mt.2012.119  1
2012 Mingozzi F, Chen Y, Murphy SL, Edmonson SC, Tai A, Price SD, Metzger ME, Zhou S, Wright JF, Donahue RE, Dunbar CE, High KA. Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 1410-6. PMID 22565846 DOI: 10.1038/mt.2012.84  1
2012 Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, McCague S, Pierce EA, Chen Y, Bennicelli JL, Zhu X, Ying GS, Sun J, Wright JF, Auricchio A, ... ... High KA, et al. AAV2 gene therapy readministration in three adults with congenital blindness. Science Translational Medicine. 4: 120ra15. PMID 22323828 DOI: 10.1126/scitranslmed.3002865  1
2012 Buchlis G, Podsakoff GM, Radu A, Hawk SM, Flake AW, Mingozzi F, High KA. Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer. Blood. 119: 3038-41. PMID 22271447 DOI: 10.1182/blood-2011-09-382317  1
2012 Wu T, Töpfer K, Lin SW, Li H, Bian A, Zhou XY, High KA, Ertl HC. Self-complementary AAVs induce more potent transgene product-specific immune responses compared to a single-stranded genome. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 572-9. PMID 22186792 DOI: 10.1038/mt.2011.280  1
2012 Cines DB, Blajchman MA, High KA, Bussel JB. Clinical trial opportunities in hemostasis and thrombosis: NHBLI State-of-the-Science symposium. American Journal of Hematology. 87: 235-8. PMID 22120890 DOI: 10.1002/ajh.22225  1
2011 Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, ... ... High KA, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. The New England Journal of Medicine. 365: 2357-65. PMID 22149959 DOI: 10.1056/NEJMoa1108046  1
2011 High KA, Aubourg P. rAAV human trial experience. Methods in Molecular Biology (Clifton, N.J.). 807: 429-57. PMID 22034041 DOI: 10.1007/978-1-61779-370-7_18  1
2011 Scola MR, Nichols TC, Zhu H, Caughey MC, Merricks EP, Raymer RA, Margaritis P, High KA, Gallippi CM. ARFI ultrasound monitoring of hemorrhage and hemostasis in vivo in canine von Willebrand disease and hemophilia. Ultrasound in Medicine & Biology. 37: 2126-32. PMID 22033127 DOI: 10.1016/j.ultrasmedbio.2011.09.006  1
2011 High KA, Skinner MW. Cell phones and landlines: the impact of gene therapy on the cost and availability of treatment for hemophilia. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 1749-50. PMID 21964303 DOI: 10.1038/mt.2011.203  1
2011 High KA. Gene therapy for haemophilia: A long and winding road Journal of Thrombosis and Haemostasis. 9: 2-11. PMID 21781236 DOI: 10.1111/j.1538-7836.2011.04369.x  1
2011 Li H, Haurigot V, Doyon Y, Li T, Wong SY, Bhagwat AS, Malani N, Anguela XM, Sharma R, Ivanciu L, Murphy SL, Finn JD, Khazi FR, Zhou S, Paschon DE, ... ... High KA, et al. In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature. 475: 217-21. PMID 21706032 DOI: 10.1038/nature10177  1
2011 Li H, Lasaro MO, Jia B, Lin SW, Haut LH, High KA, Ertl HC. Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 2021-30. PMID 21587208 DOI: 10.1038/mt.2011.81  1
2011 Mingozzi F, High KA. Immune responses to AAV in clinical trials. Current Gene Therapy. 11: 321-30. PMID 21557723 DOI: 10.2174/156652311796150354  1
2011 Bish LT, Sleeper MM, Reynolds C, Gazzara J, Withnall E, Singletary GE, Buchlis G, Hui D, High KA, Gao G, Wilson JM, Sweeney HL. Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines. Human Gene Therapy. 22: 969-77. PMID 21542669 DOI: 10.1089/hum.2011.035  1
2011 Mingozzi F, High KA. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nature Reviews. Genetics. 12: 341-55. PMID 21499295 DOI: 10.1038/nrg2988  1
2011 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: 3974-82. PMID 21325603 DOI: 10.1182/blood-2010-09-309732  1
2011 Zhou J, Yang X, Wright JF, High KA, Couto L, Qu G. PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9. Journal of Virological Methods. 173: 99-107. PMID 21295608 DOI: 10.1016/j.jviromet.2011.01.013  1
2011 Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, et al. Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 876-85. PMID 21245849 DOI: 10.1038/mt.2010.274  1
2011 Li H, Tuyishime S, Wu TL, Giles-Davis W, Zhou D, Xiao W, High KA, Ertl HC. Adeno-associated virus vectors serotype 2 induce prolonged proliferation of capsid-specific CD8+ T cells in mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 536-46. PMID 21157435 DOI: 10.1038/mt.2010.267  1
2011 Favaro P, Finn JD, Siner JI, Wright JF, High KA, Arruda VR. Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model. Human Gene Therapy. 22: 843-52. PMID 21126217 DOI: 10.1089/hum.2010.155  1
2011 Li H, Malani N, Hamilton SR, Schlachterman A, Bussadori G, Edmonson SE, Shah R, Arruda VR, Mingozzi F, Wright JF, Bushman FD, High KA. Assessing the potential for AAV vector genotoxicity in a murine model. Blood. 117: 3311-9. PMID 21106988 DOI: 10.1182/blood-2010-08-302729  1
2011 Scola MR, Baggesen LM, Behler RH, Nichols TC, Zhu H, Caughey MC, Merricks EP, Raymer RA, Margaritis P, High KA, Gallippi CM. In vivo detection of hemorrhage rate in dog models of hemophilia and VWD and at human femoral arteriotomy by ARFI ultrasound Ieee International Ultrasonics Symposium, Ius. 660-663. DOI: 10.1109/ULTSYM.2011.0160  1
2010 Wright JF, Wellman J, High KA. Manufacturing and regulatory strategies for clinical AAV2-hRPE65. Current Gene Therapy. 10: 341-9. PMID 20712582  1
2010 Taylor PL, Barker RA, Blume KG, Cattaneo E, Colman A, Deng H, Edgar H, Fox IJ, Gerstle C, Goldstein LS, High KA, Lyall A, Parkman R, Pitossi FJ, Prentice ED, et al. Patients beware: commercialized stem cell treatments on the web. Cell Stem Cell. 7: 43-9. PMID 20621049 DOI: 10.1016/j.stem.2010.06.001  1
2010 Couto LB, High KA. Viral vector-mediated RNA interference. Current Opinion in Pharmacology. 10: 534-42. PMID 20620113 DOI: 10.1016/j.coph.2010.06.007  1
2010 Batorova A, High KA, Gringeri A. Special lectures in haemophilia management Haemophilia. 16: 22-28. PMID 20590852 DOI: 10.1111/j.1365-2516.2010.02289.x  1
2010 Margaritis P, High KA. Gene therapy in haemophilia - going for cure? Haemophilia. 16: 24-28. PMID 20586798 DOI: 10.1111/j.1365-2516.2010.02256.x  1
2010 Nichols TC, Raymer RA, Franck HW, Merricks EP, Bellinger DA, DeFriess N, Margaritis P, Arruda VR, Kay MA, High KA. Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B. Haemophilia : the Official Journal of the World Federation of Hemophilia. 16: 19-23. PMID 20586797 DOI: 10.1111/j.1365-2516.2010.02255.x  1
2010 Lin CN, Kao CY, Miao CH, Hamaguchi N, Wu HL, Shi GY, Liu YL, High KA, Lin SW. Generation of a novel factor IX with augmented clotting activities in vitro and in vivo. Journal of Thrombosis and Haemostasis : Jth. 8: 1773-83. PMID 20492477 DOI: 10.1111/j.1538-7836.2010.03913.x  1
2010 Haurigot 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. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 1318-29. PMID 20424599 DOI: 10.1038/mt.2010.73  1
2010 Amado D, Mingozzi F, Hui D, Bennicelli JL, Wei Z, Chen Y, Bote E, Grant RL, Golden JA, Narfstrom K, Syed NA, Orlin SE, High KA, Maguire AM, Bennett J. Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness. Science Translational Medicine. 2: 21ra16. PMID 20374996 DOI: 10.1126/scitranslmed.3000659  1
2010 Arruda VR, Stedman HH, Haurigot V, Buchlis G, Baila S, Favaro P, Chen Y, Franck HG, Zhou S, Wright JF, Couto LB, Jiang H, Pierce GF, Bellinger DA, Mingozzi F, ... ... High KA, et al. Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. Blood. 115: 4678-88. PMID 20335222 DOI: 10.1182/blood-2009-12-261156  1
2010 Ayuso E, Mingozzi F, Montane J, Leon X, Anguela XM, Haurigot V, Edmonson SA, Africa L, Zhou S, High KA, Bosch F, Wright JF. High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency. Gene Therapy. 17: 503-10. PMID 19956269 DOI: 10.1038/gt.2009.157  1
2010 Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, Rossi S, Marshall K, Banfi S, Surace EM, Sun J, Redmond TM, Zhu X, Shindler KS, Ying GS, ... ... High KA, et al. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 643-50. PMID 19953081 DOI: 10.1038/mt.2009.277  1
2010 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. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 135-42. PMID 19904235 DOI: 10.1038/mt.2009.257  1
2010 Scola MR, Gallippi CM, Nichols TC, Caughey MC, Margaritis P, High KA. ARFI ultrasound for in vivo monitoring of soft-tissue bleeding and hemostasis in a dog model of hemophilia Proceedings - Ieee Ultrasonics Symposium. 1149-1152. DOI: 10.1109/ULTSYM.2010.5935645  1
2010 High KA, Wright JF. Gene Therapy Trials in Hemophilia A and B Textbook of Hemophilia: Second Edition. 231-238. DOI: 10.1002/9781444318555.ch36  1
2009 Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JI, et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet. 374: 1597-605. PMID 19854499 DOI: 10.1016/S0140-6736(09)61836-5  1
2009 Mingozzi F, High KA. Gateway to the diseased brain. Nature Medicine. 15: 1123-4. PMID 19812565 DOI: 10.1038/nm1009-1123  1
2009 High KA. The Jeremiah Metzger Lecture: gene therapy for inherited disorders: from Christmas disease to Leber's amaurosis. Transactions of the American Clinical and Climatological Association. 120: 331-59. PMID 19768188  1
2009 Cao O, Hoffman BE, Moghimi B, Nayak S, Cooper M, Zhou S, Ertl HC, High KA, Herzog RW. Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1733-42. PMID 19603001 DOI: 10.1038/mt.2009.159  1
2009 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. 114: 2077-86. PMID 19506302 DOI: 10.1182/blood-2008-07-167510  1
2009 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. The Journal of Clinical Investigation. 119: 1688-95. PMID 19436115 DOI: 10.1172/JCI36891  1
2009 Li H, Lin SW, Giles-Davis W, Li Y, Zhou D, Xiang ZQ, High KA, Ertl HC. A preclinical animal model to assess the effect of pre-existing immunity on AAV-mediated gene transfer. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1215-24. PMID 19367258 DOI: 10.1038/mt.2009.79  1
2009 Favaro P, Downey HD, Zhou JS, Wright JF, Hauck B, Mingozzi F, High KA, Arruda VR. Host and vector-dependent effects on the risk of germline transmission of AAV vectors. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 1022-30. PMID 19293773 DOI: 10.1038/mt.2009.56  1
2009 Nichols TC, Dillow AM, Franck HW, Merricks EP, Raymer RA, Bellinger DA, Arruda VR, High KA. Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency. Ilar Journal / National Research Council, Institute of Laboratory Animal Resources. 50: 144-67. PMID 19293459  1
2009 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. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 417-24. PMID 19127250 DOI: 10.1038/mt.2008.292  1
2009 Margaritis P, Roy E, Aljamali MN, Downey HD, Giger U, Zhou S, Merricks E, Dillow A, Ezban M, Nichols TC, High KA. Successful treatment of canine hemophilia by continuous expression of canine FVIIa. Blood. 113: 3682-9. PMID 19109232 DOI: 10.1182/blood-2008-07-168377  1
2009 Murphy SL, Li H, Mingozzi F, Sabatino DE, Hui DJ, Edmonson SA, High KA. Diverse IgG subclass responses to adeno-associated virus infection and vector administration. Journal of Medical Virology. 81: 65-74. PMID 19031458 DOI: 10.1002/jmv.21360  1
2009 Gupta S, Kühnisch J, Mustafa A, Lhotak S, Schlachterman A, Slifker MJ, Klein-Szanto A, High KA, Austin RC, Kruger WD. Mouse models of cystathionine beta-synthase deficiency reveal significant threshold effects of hyperhomocysteinemia. Faseb Journal : Official Publication of the Federation of American Societies For Experimental Biology. 23: 883-93. PMID 18987302 DOI: 10.1096/fj.08-120584  1
2009 Niemeyer GP, Herzog RW, Mount J, Arruda VR, Tillson DM, Hathcock J, van Ginkel FW, High KA, Lothrop CD. Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy. Blood. 113: 797-806. PMID 18957684 DOI: 10.1182/blood-2008-10-181479  1
2009 Hauck B, Murphy SL, 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 preformed capsid in immune responses. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 144-52. PMID 18941440 DOI: 10.1038/mt.2008.227  1
2008 Murphy SL, Li H, Zhou S, Schlachterman A, High KA. Corrigendum to "Prolonged Susceptibility to Antibody-mediated Neutralization for Adeno-associated Vectors Targeted to the Liver". Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 633. PMID 28178504 DOI: 10.1038/sj.mt.6300386  1
2008 Toromanoff A, Chérel Y, Guilbaud M, Penaud-Budloo M, Snyder RO, Haskins ME, Deschamps JY, Guigand L, Podevin G, Arruda VR, High KA, Stedman HH, Rolling F, Anegon I, Moullier P, et al. Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 1291-1299. PMID 28178483 DOI: 10.1038/mt.2008.87  1
2008 Hyun I, Lindvall O, Ahrlund-Richter L, Cattaneo E, Cavazzana-Calvo M, Cossu G, De Luca M, Fox IJ, Gerstle C, Goldstein RA, Hermerén G, High KA, Kim HO, Lee HP, Levy-Lahad E, et al. New ISSCR guidelines underscore major principles for responsible translational stem cell research. Cell Stem Cell. 3: 607-9. PMID 19041777 DOI: 10.1016/j.stem.2008.11.009  1
2008 Murphy SL, Bhagwat A, Edmonson S, Zhou S, High KA. High-throughput screening and biophysical interrogation of hepatotropic AAV. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 1960-7. PMID 18827805 DOI: 10.1038/mt.2008.210  1
2008 Stroes ES, Nierman MC, Meulenberg JJ, Franssen R, Twisk J, Henny CP, Maas MM, Zwinderman AH, Ross C, Aronica E, High KA, Levi MM, Hayden MR, Kastelein JJ, Kuivenhoven JA. Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arteriosclerosis, Thrombosis, and Vascular Biology. 28: 2303-4. PMID 18802015 DOI: 10.1161/ATVBAHA.108.175620  1
2008 Pipe SW, High KA, Ohashi K, Ural AU, Lillicrap D. Progress in the molecular biology of inherited bleeding disorders Haemophilia. 14: 130-137. PMID 18510533 DOI: 10.1111/j.1365-2516.2008.01718.x  1
2008 Toromanoff A, Chérel Y, Guilbaud M, Penaud-Budloo M, Snyder RO, Haskins ME, Deschamps JY, Guigand L, Podevin G, Arruda VR, High KA, Stedman HH, Rolling F, Anegon I, Moullier P, et al. Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 1291-9. PMID 18461055 DOI: 10.1038/mt.2008.87  1
2008 Maguire AM, Simonelli F, Pierce EA, Pugh EN, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, ... ... High KA, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. The New England Journal of Medicine. 358: 2240-8. PMID 18441370 DOI: 10.1056/NEJMoa0802315  1
2008 Hasbrouck NC, High KA. AAV-mediated gene transfer for the treatment of hemophilia B: Problems and prospects Gene Therapy. 15: 870-875. PMID 18432276 DOI: 10.1038/gt.2008.71  1
2008 Aljamali MN, Margaritis P, Schlachterman A, Tai SJ, Roy E, Bunte R, Camire RM, High KA. Long-term expression of murine activated factor VII is safe, but elevated levels cause premature mortality. The Journal of Clinical Investigation. 118: 1825-34. PMID 18398505 DOI: 10.1172/JCI32878  1
2008 Murphy SL, High KA. Gene therapy for haemophilia. British Journal of Haematology. 140: 479-87. PMID 18275425 DOI: 10.1111/j.1365-2141.2007.06942.x  1
2008 Bennicelli J, Wright JF, Komaromy A, Jacobs JB, Hauck B, Zelenaia O, Mingozzi F, Hui D, Chung D, Rex TS, Wei Z, Qu G, Zhou S, Zeiss C, Arruda VR, ... ... High KA, et al. Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 458-65. PMID 18209734 DOI: 10.1038/sj.mt.6300389  1
2008 Tai SJ, Herzog RW, Margaritis P, 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 of Thrombosis and Haemostasis : Jth. 6: 339-45. PMID 18036190 DOI: 10.1111/j.1538-7836.2007.02849.x  1
2008 Murphy SL, Li H, Zhou S, Schlachterman A, High KA, High K. Prolonged susceptibility to antibody-mediated neutralization for adeno-associated vectors targeted to the liver. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 138-45. PMID 17955024 DOI: 10.1038/sj.mt.6300334  1
2007 High KA. Update on progress and hurdles in novel genetic therapies for hemophilia. Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program. 466-72. PMID 18024666 DOI: 10.1182/asheducation-2007.1.466  1
2007 Mingozzi F, High KA. Immune responses to AAV in clinical trials. Current Gene Therapy. 7: 316-24. PMID 17979678 DOI: 10.2174/156652307782151425  1
2007 Yang C, Feng J, Song W, Wang J, Tsai B, Zhang Y, Scaringe WA, Hill KA, Margaritis P, High KA, Sommer SS. A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin. Proceedings of the National Academy of Sciences of the United States of America. 104: 15394-9. PMID 17881586 DOI: 10.1073/pnas.0610878104  1
2007 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 nonhuman primate model of AAV-mediated gene transfer to liver. Blood. 110: 2334-41. PMID 17609423 DOI: 10.1182/blood-2007-03-080093  1
2007 Sabatino DE, Mackenzie TC, Peranteau W, Edmonson S, Campagnoli C, Liu YL, Flake AW, High KA. Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 1677-85. PMID 17565352 DOI: 10.1038/sj.mt.6300219  1
2007 Ingerslev J, Herlin T, Sørensen 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 : the Official Journal of the World Federation of Hemophilia. 13: 334-6. PMID 17498086 DOI: 10.1111/j.1365-2516.2007.01466.x  1
2007 Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, Rasko JE, Ragni MV, Manno CS, Sommer J, Jiang H, Pierce GF, Ertl HC, High KA. CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nature Medicine. 13: 419-22. PMID 17369837 DOI: 10.1038/nm1549  1
2007 Li H, Murphy SL, Giles-Davis W, Edmonson S, Xiang Z, Li Y, Lasaro MO, High KA, Ertl HC. Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 792-800. PMID 17245353 DOI: 10.1038/sj.mt.6300090  1
2007 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, et al. Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography. Journal of Virological Methods. 140: 183-92. PMID 17196264 DOI: 10.1016/j.jviromet.2006.11.019  1
2006 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. Journal of Thrombosis and Haemostasis : Jth. 4: 2616-22. PMID 16961583 DOI: 10.1111/j.1538-7836.2006.02203.x  1
2006 Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy D, Vargas JA, Zhou S, Scallan CD, Sommer J, Vijay S, Mingozzi F, High KA, Pierce GF. Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood. 108: 3321-8. PMID 16868252 DOI: 10.1182/blood-2006-04-017913  1
2006 High KA. The leak stops here: platelets as delivery vehicles for coagulation factors. The Journal of Clinical Investigation. 116: 1840-2. PMID 16823486 DOI: 10.1172/JCI29193  1
2006 Jiang H, Pierce GF, Ozelo MC, de Paula EV, Vargas JA, Smith P, Sommer J, Luk A, Manno CS, High KA, Arruda VR. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Molecular Therapy : the Journal of the American Society of Gene Therapy. 14: 452-5. PMID 16822719 DOI: 10.1016/j.ymthe.2006.05.004  1
2006 Cao O, Armstrong E, Schlachterman A, Wang L, Okita DK, Conti-Fine B, High KA, Herzog RW. Immune deviation by mucosal antigen administration suppresses gene-transfer-induced inhibitor formation to factor IX. Blood. 108: 480-6. PMID 16543469 DOI: 10.1182/blood-2005-11-4668  1
2006 Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Rasko J, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, ... ... High KA, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nature Medicine. 12: 342-7. PMID 16474400 DOI: 10.1038/nm1358  1
2006 Margaritis P, High KA. Advances in gene therapy using factor VIIa in hemophilia. Seminars in Hematology. 43: S101-4. PMID 16427374 DOI: 10.1053/j.seminhematol.2005.11.015  1
2005 Sabatino DE, Mingozzi F, Hui DJ, Chen H, Colosi P, Ertl HC, High KA. Identification of mouse AAV capsid-specific CD8+ T cell epitopes. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 1023-33. PMID 16263332 DOI: 10.1016/j.ymthe.2005.09.009  1
2005 High KA. Stakeholders' conference sharpens focus on challenges of clinical gene transfer. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 581-2. PMID 16140585 DOI: 10.1016/j.ymthe.2005.08.015  1
2005 High KA. Gene therapy: the moving finger. Nature. 435: 577-9. PMID 15931206 DOI: 10.1038/435577a  1
2005 Zhang HG, High KA, Wu Q, Yang P, Schlachterman A, Yu S, Yi N, Hsu HC, Mountz JD. Genetic analysis of the antibody response to AAV2 and factor IX. Molecular Therapy : the Journal of the American Society of Gene Therapy. 11: 866-74. PMID 15922957 DOI: 10.1016/j.ymthe.2005.02.014  1
2005 Arruda VR, Stedman HH, Nichols TC, Haskins ME, Nicholson M, Herzog RW, Couto LB, High KA. Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood. 105: 3458-64. PMID 15479726 DOI: 10.1182/blood-2004-07-2908  1
2004 Liu YL, Mingozzi F, Rodriguéz-Colôn SM, Joseph S, 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. Human Gene Therapy. 15: 783-92. PMID 15319035 DOI: 10.1089/1043034041648453  1
2004 Whinna HC, Lesesky EB, Monroe DM, High KA, Larson PJ, Church FC. Role of the γ-carboxyglutamic acid domain of activated factor X in the presence of calcium during inhibition by antithrombin-heparin Journal of Thrombosis and Haemostasis. 2: 1127-1134. PMID 15219196 DOI: 10.1111/j.1538-7836.2004.00796.x  1
2004 Sabatino DE, Armstrong E, Edmonson S, Liu YL, 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: 2767-74. PMID 15217833 DOI: 10.1182/blood-2004-03-1028  1
2004 High KA. Clinical gene transfer studies for hemophilia B. Seminars in Thrombosis and Hemostasis. 30: 257-67. PMID 15118937 DOI: 10.1055/s-2004-825639  1
2004 Margaritis P, Arruda VR, Aljamali M, Camire RM, Schlachterman A, High KA. Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII. The Journal of Clinical Investigation. 113: 1025-31. PMID 15057309 DOI: 10.1172/JCI20106  1
2004 Arruda VR, Schuettrumpf J, Herzog RW, Nichols TC, Robinson N, Lotfi 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. PMID 12969984 DOI: 10.1182/blood-2003-05-1446  1
2003 High KA. Coaxing coagulation with RNA and cell fragments. Nature Medicine. 9: 991-2. PMID 12894156 DOI: 10.1038/nm0803-991  1
2003 High KA. Theodore E. Woodward Award. AAV-mediated gene transfer for hemophilia. Transactions of the American Clinical and Climatological Association. 114: 337-51; discussion 3. PMID 12813929  1
2003 Gerecitano J, Mathias 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 1+2 levels in patients with veno-occlusive disease after stem cell transplantation. Journal of Hematotherapy & Stem Cell Research. 12: 215-23. PMID 12804180 DOI: 10.1089/152581603321628359  1
2003 High KA. The risks of germline gene transfer. The Hastings Center Report. 33: 3. PMID 12760106  1
2003 Mingozzi F, Liu YL, Dobrzynski E, Kaufhold A, Liu JH, Wang Y, Arruda VR, High KA, Herzog RW. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. The Journal of Clinical Investigation. 111: 1347-56. PMID 12727926 DOI: 10.1172/JCI16887  1
2003 Fraser R, Le Couteur DG, Warren A, Cogger VC, Bertolino P, Smith M, High KA, Herzog R, Arruda V. The liver sieve and gene therapy [2] (multiple letters) Blood. 101: 3338-3339. PMID 12672697 DOI: 10.1182/blood-2002-12-3657  1
2003 High KA. Gene transfer as an approach to treating hemophilia. Seminars in Thrombosis and Hemostasis. 29: 107-20. PMID 12640573 DOI: 10.1055/s-2003-37945  1
2003 Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DG, Johnson FA, McClelland A, Scallan C, ... ... High KA, et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood. 101: 2963-72. PMID 12515715 DOI: 10.1182/blood-2002-10-3296  1
2003 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 Biochemical Journal. 369: 563-571. PMID 12358603 DOI: 10.1042/BJ20020888  1
2002 High KA. Adeno-associated virus-mediated gene transfer for hemophilia B International Journal of Hematology. 76: 310-318. PMID 12463593 DOI: 10.1007/BF02982689  1
2002 Mingozzi F, Schüttrumpf J, Arruda VR, Liu Y, Liu YL, High KA, Xiao W, Herzog RW. Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector. Journal of Virology. 76: 10497-502. PMID 12239326  1
2002 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. Human Gene Therapy. 13: 1281-91. PMID 12162811 DOI: 10.1089/104303402760128513  1
2002 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 Biochemical Journal. 363: 411-416. PMID 11931672 DOI: 10.1042/0264-6021:3630411  1
2002 Mount JD, Herzog RW, Tillson DM, Goodman SA, Robinson N, McCleland ML, Bellinger D, Nichols TC, Arruda VR, Lothrop CD, High KA. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood. 99: 2670-6. PMID 11929752  1
2001 High KA. AAV-mediated gene transfer for hemophilia Annals of the New York Academy of Sciences. 953: 64-74. PMID 11795424  1
2001 Arruda VR, Fields PA, Milner R, Wainwright L, De Miguel MP, Donovan PJ, Herzog RW, Nichols TC, Biegel JA, Razavi M, Dake M, Huff D, Flake AW, Couto L, Kay MA, ... High KA, et al. Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males. Molecular Therapy : the Journal of the American Society of Gene Therapy. 4: 586-92. PMID 11735343 DOI: 10.1006/mthe.2001.0491  1
2001 Larson PJ, High KA. Gene therapy for hemophilia B: AAV-mediated transfer of the gene for coagulation factor IX to human muscle Advances in Experimental Medicine and Biology. 489: 45-57. PMID 11554590  1
2001 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 F9 Molecular Therapy. 4: 201-210. PMID 11545610 DOI: 10.1006/mthe.2001.0441  1
2001 Herzog RW, Mount JD, Arruda VR, High KA, Lothrop CD. 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. PMID 11545609 DOI: 10.1006/mthe.2001.0442  1
2001 Fields PA, 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 factor IX in C57BL/6 mice. Gene Therapy. 8: 354-61. PMID 11313811 DOI: 10.1038/sj.gt.3301409  1
2001 High KA. Gene therapy: A 2001 perspective Haemophilia. 7: 23-27. PMID 11240615 DOI: 10.1046/j.1365-2516.2001.00098.x  1
2001 Hung HL, Pollak ES, Kudaravalli RD, Arruda V, Chu K, High KA. Regulation of human coagulation factor X gene expression by GATA-4 and the Sp family of transcription factors Blood. 97: 946-951. PMID 11159521 DOI: 10.1182/blood.V97.4.946  1
2001 Arruda VR, Hagstrom JN, Deitch J, Heiman-Patterson T, Camire RM, Chu K, Fields PA, Herzog RW, Couto LB, Larson PJ, High KA. Posttranslational modifications of recombinant myotube-synthesized human factor IX Blood. 97: 130-138. PMID 11133752 DOI: 10.1182/blood.V97.1.130  1
2000 High KA. Gene therapy in haematology and oncology Lancet. 356. PMID 11191528 DOI: 10.1016/S0140-6736(00)91994-9  1
2000 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. PMID 11087381 DOI: 10.1021/bi001074q  1
2000 Herzog RW, Arruda VR, Fisher TH, Read MS, Nichols TC, High KA. Absence of circulating factor IX antigen in hemophilia B dogs of the UNC-Chapel Hill colony Thrombosis and Haemostasis. 84: 352-354. PMID 10959714  1
2000 Fields PA, Kowalczyk DW, Arruda VR, Armstrong E, McCleland ML, Hagstrom JN, Pasi KJ, Ertl HCJ, Herzog RW, High KA. Role of Vector in Activation of T Cell Subsets in Immune Responses against the Secreted Transgene Product Factor IX Molecular Therapy. 1: 225-235. PMID 10933938 DOI: 10.1006/mthe.2000.0032  1
2000 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. PMID 10753832  1
2000 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, ... High KA, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nature Genetics. 24: 257-61. PMID 10700178 DOI: 10.1038/73464  1
1999 Herzog RW, High KA. Adeno-associated virus-mediated gene transfer of factor IX for treatment of hemophilia B by gene therapy Thrombosis and Haemostasis. 82: 540-546. PMID 10605748  1
1999 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. PMID 10569955 DOI: 10.1021/bi991544k  1
1999 Herzog RW, Yang EY, Couto LB, Hagstrom JN, Elwell D, Fields PA, Burton M, Bellinger DA, 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. PMID 9883840 DOI: 10.1038/4743  1
1998 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 Journal of Pediatrics. 133: 777-781. PMID 9842043  1
1998 Herzog RW, High KA. Problems and prospects in gene therapy for hemophilia Current Opinion in Hematology. 5: 321-326. PMID 9776210  1
1998 Carew JA, Pollak ES, High KA, Bauer KA. Severe factor VII deficiency due to a mutation disrupting an Sp1 binding site in the factor VII promoter Blood. 92: 1639-1645. PMID 9716591  1
1998 Nakai H, Herzog RW, Hagstrom JN, Walter J, Kung SH, Yang EY, Tai SJ, Iwaki Y, Kurtzman GJ, Fisher KJ, Colosi P, Couto LB, High KA. Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood. 91: 4600-7. PMID 9616156  1
1998 Larson PJ, Camire RM, 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. PMID 9538022 DOI: 10.1021/bi972428p  1
1998 Kung SH, Hagstrom JN, Cass D, Tai SJ, Lin HF, Stafford DW, High KA. Human factor IX corrects the bleeding diathesis of mice with hemophilia B. Blood. 91: 784-90. PMID 9446637  1
1997 Walter J, High KA. Gene therapy for the hemophilias Advances in Veterinary Medicine. 40: 119-134. PMID 9395731 DOI: 10.1016/S0065-3519(97)80006-7  1
1997 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 gamma-glutamyl carboxylase. Blood. 89: 4058-62. PMID 9166845  1
1997 Herzog RW, Hagstrom JN, Kung SH, Tai SJ, Wilson JM, Fisher KJ, High KA. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proceedings of the National Academy of Sciences of the United States of America. 94: 5804-9. PMID 9159155 DOI: 10.1073/pnas.94.11.5804  1
1997 Ambrosini G, Plescia J, Chu KC, High KA, Altieri DC. Activation-dependent exposure of the inter-EGF sequence Leu83-Leu88 in factor Xa mediates ligand binding to effector cell protease receptor-1. The Journal of Biological Chemistry. 272: 8340-5. PMID 9079657 DOI: 10.1074/jbc.272.13.8340  1
1997 Arbini AA, Pollak ES, Bayleran JK, High KA, 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. PMID 8978290  1
1996 Sridhara S, Chaing S, High KA, Blajchman MA, Clarke BJ. Activation of a recombinant human factor VII structural analogue alters its affinity of binding to tissue factor American Journal of Hematology. 53: 66-71. PMID 8892729 DOI: 10.1002/(SICI)1096-8652(199610)53:2<66::AID-AJH2>3.0.CO;2-1  1
1996 Chu K, Wu SM, Stanley T, Stafford DW, High KA. A mutation in the propeptide of factor IX leads to warfarin sensitivity by a novel mechanism Journal of Clinical Investigation. 98: 1619-1625. PMID 8833911  1
1996 Larson PJ, 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 Journal of Biological Chemistry. 271: 3869-3876. PMID 8632006 DOI: 10.1074/jbc.271.7.3869  1
1996 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 Proceedings of the National Academy of Sciences of the United States of America. 93: 3056-3061. PMID 8610167 DOI: 10.1073/pnas.93.7.3056  1
1996 Pollak ES, Hung HL, Godin W, Overton GC, High KA. Functional characterization of the human factor VII 5′-flanking region Journal of Biological Chemistry. 271: 1738-1747. PMID 8576177  1
1996 Hung HL, High KA. Liver-enriched transcription factor HNF-4 and ubiquitous factor NF-Y are critical for expression of blood coagulation factor X Journal of Biological Chemistry. 271: 2323-2331. PMID 8567696 DOI: 10.1074/jbc.271.4.2323  1
1996 Pollak ES, Hung HL, High KA. Critical elements in the human factor vu promoter region Faseb Journal. 10.  1
1995 High KA. Factor IX: Molecular structure, epitopes, and mutations associated with inhibitor formation Advances in Experimental Medicine and Biology. 386: 79-86. PMID 8851016  1
1995 Bi L, Lawler AM, Antonarakis SE, High KA, Gearhart JD, Kazazian HH. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A Nature Genetics. 10: 119-121. PMID 7647782 DOI: 10.1038/ng0595-119  1
1994 Chaing S, Clarke B, Sridhara S, Chu K, Friedman P, VanDusen W, Roberts HR, Blajchman M, Monroe DM, High KA. Severe factor VII deficiency caused by mutations abolishing the cleavage site for activation and altering binding to tissue factor Blood. 83: 3524-3535. PMID 8204879  1
1994 Lozier JN, Thompson AR, Hu PC, Read M, Brinkhous KM, High KA, Curiel DT. Efficient transfection of primary cells in a canine hemophilia B model using adenovirus-polylysine-DNA complexes Human Gene Therapy. 5: 313-322. PMID 8018746  1
1994 Chaing SH, Wallmark A, Berntorp E, High KA. A NlaIII polymorphism within the human factor VII gene Human Genetics. 93: 722-723. PMID 8005605 DOI: 10.1007/BF00201584  1
1993 Rose VL, Dermott SC, Murray BF, McIver MM, High KA, Oberhardt BJ. Decentralized testing for prothrombin time and activated partial thromboplastin time using a dry chemistry portable analyzer Archives of Pathology and Laboratory Medicine. 117: 611-617. PMID 8503733  1
1993 Racchi M, Watzke HH, High KA, 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 Journal of Biological Chemistry. 268: 5735-5740. PMID 8449937  1
1993 Giannelli F, Green PM, High KA, Sommer S, Poon MC, Ludwig M, Schwaab R, Reitsma PH, Goossens M, Yoshioka A. Haemophilia B: database of point mutations and short additions and deletions--fourth edition, 1993. Nucleic Acids Research. 21: 3075-87. PMID 8392713 DOI: 10.1093/nar/21.13.3075  1
1993 Sridhara S, Clarke BJ, Ofosu FA, High KA, Blajchman MA. The direct binding of human factor VII in plasma to recombinant human tissue factor Thrombosis Research. 70: 307-316. PMID 8332960 DOI: 10.1016/0049-3848(93)90103-U  1
1992 Giannelli F, Green PM, High KA, Sommer S, Lillicrap DP, Ludwig M, Olek K, Reitsma PH, Goossens M, Yoshioka A, Brownlee GG. Haemophilia B: Database of point mutations and short additions and deletions - Third edition, 1992 Nucleic Acids Research. 20: 2027-2063. PMID 1598234  1
1992 Larson PJ, High KA. Biology of inherited coagulopathies: Factor IX Hematology/Oncology Clinics of North America. 6: 999-1009. PMID 1356960  1
1992 Huang MN, Hung HL, Stanfield-Oakley SA, High KA. Characterization of the human blood coagulation Factor X promoter Journal of Biological Chemistry. 267: 15440-15446. PMID 1322399  1
1991 Watzke HH, Wallmark A, Hamaguchi N, Giardina P, Stafford DW, High KA. Factor XSanto Domingo evidence that the severe clinical phenotype arises from a mutation blocking secretion Journal of Clinical Investigation. 88: 1685-1689. PMID 1939653  1
1991 Wallmark A, Rose VL, Ho C, High KA. A NlalV polymorphism within the human factor X gene Nucleic Acids Research. 19: 4022. PMID 1677764 DOI: 10.1093/nar/19.14.4022-a  1
1990 Lozier JN, Monroe DM, Stanfield-Oakley S, Lin SW, Smith KJ, Roberts HR, High KA. Factor IX New London: Substitution of proline for glutamine at position 50 causes severe hemophilia B Blood. 75: 1097-1104. PMID 2306516  1
1990 Huang MN, High KA. Efficient subcloning of DNA fragments amplified by crude oligonucleotides Biotechniques. 9: 710-711. PMID 2271170  1
1990 Lozier JN, High KA. Molecular basis of hemophilia Hematologic Pathology. 4: 1-26. PMID 2111315  1
1990 Watzke HH, Lechner K, Roberts HR, Reddy SV, Welsch DJ, Friedman P, Mahr G, Jagadeeswaran P, Monroe DM, High KA. Molecular defect (Gla+14→Lys) and its functional consequences in a hereditary factor X deficiency (factor X "Vorarlberg") Journal of Biological Chemistry. 265: 11982-11989. PMID 1973167  1
1989 Reddy SV, Zhou ZQ, Rao KJ, Scott JP, Watzke H, High KA, Jagadeeswaran P. Molecular characterization of human factor XSan Antonio Blood. 74: 1486-1490. PMID 2790181  1
1989 Evans JP, Watzke HH, Ware JL, Stafford DW, High KA. Molecular cloning of a cDNA encoding canine factor IX Blood. 74: 207-212. PMID 2752110  1
1989 Monroe DM, McCord DM, Huang MN, High KA, Lundblad RL, Kasper CK, Roberts HR. Functional consequences of an arginine180 to glutamine mutation in factor IX Hilo Blood. 73: 1540-1544. PMID 2713493  1
1989 Dent GA, Ayscue LH, High KA, Leglise MC, 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. PMID 2676187  1
1989 Hung MN, Kasper CK, Roberts HR, Stafford DW, High KA. Molecular defect in factor IX(Hilo), a hemophilia B(m) variant: Arg → Gln at the carboxyterminal cleavage site of the activation peptide Blood. 73: 718-721. PMID 2563663  1
1989 Evans JP, Brinkhous KM, Brayer GD, Reisner HM, High KA. Canine hemophilia B resulting from a point mutation with unusual consequences Proceedings of the National Academy of Sciences of the United States of America. 86: 10095-10099. PMID 2481310 DOI: 10.1073/pnas.86.24.10095  1
1987 High KA, Stolle CA, Schneider JW, Hu W, Benz EJ. c-myc Gene inactivation during induced maturation of HL-60 cells. Transcriptional repression and loss of a specific DNAse I hypersensitive site Journal of Clinical Investigation. 79: 93-99. PMID 3540012  1
1985 Lambert SR, High KA, Benz EJ, Cotlier E. Serous Retinal Detachments in Thrombotic Thrombocytopenic Purpura Archives of Ophthalmology. 103: 1172-1174. PMID 4040743 DOI: 10.1001/archopht.1985.01050080084026  1
1980 Weinstein PS, High KA, D'Ercole AJ, Jennette JC. Insulin resistance due to receptor antibodies: A complication of progressive systemic sclerosis Arthritis and Rheumatism. 23: 101-105. PMID 7352930  1
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