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Katherine A. High - Publications

Affiliations: 
Cell and Molecular Biology University of Pennsylvania, Philadelphia, PA, United States 
Area:
Immunology, Cell Biology, Molecular Biology
Website:
http://www.med.upenn.edu/apps/faculty/index.php/g275/p12112
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295 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
2023 High KA. THE JEREMIAH METZGER LECTURE: TURNING GENES INTO MEDICINES: HIGHLIGHTS AND HURDLES IN THE DEVELOPMENT OF GENE THERAPY FOR GENETIC DISEASE. Transactions of the American Clinical and Climatological Association. 133: 204-233. PMID 37701622  0.392
2022 Elkouby L, Armour SM, Toso R, DiPietro M, Davidson RJ, Nguyen GN, Willet M, Kutza S, Silverberg J, Frick J, Crosariol M, Wang Y, Wang C, High KA, Sabatino DE, et al. Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A. Molecular Therapy. Methods & Clinical Development. 24: 20-29. PMID 34977269 DOI: 10.1016/j.omtm.2021.11.005  0.413
2021 George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, ... ... High KA, et al. Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A. The New England Journal of Medicine. 385: 1961-1973. PMID 34788507 DOI: 10.1056/NEJMoa2104205  0.345
2020 Zou C, Vercauteren KOA, Michailidis E, Kabbani M, Zoluthkin I, Quirk C, Chiriboga L, Yazicioglu M, Anguela XM, Meuleman P, High KA, Herzog RW, de Jong YP. Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice. Molecular Therapy. Methods & Clinical Development. 18: 189-198. PMID 32637450 DOI: 10.1016/J.Omtm.2020.05.033  0.614
2020 George LA, Ragni MV, Rasko JEJ, Raffini LJ, Samelson-Jones BJ, Ozelo M, Hazbon M, Runowski AR, Wellman JA, Wachtel K, Chen Y, Anguela XM, Kuranda K, Mingozzi F, High KA. Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 32559433 DOI: 10.1016/J.Ymthe.2020.06.001  0.505
2019 Xiang Z, Kurupati RK, Li Y, Kuranda K, Zhou X, Mingozzi F, High KA, Ertl HCJ. The Effect of CpG Sequences on Capsid-Specific CD8 T Cell Responses to AAV Vector Gene Transfer. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 31839483 DOI: 10.1016/J.Ymthe.2019.11.014  0.402
2019 Maguire AM, Russell S, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, Marshall KA, McCague S, Reichert H, Davis M, Simonelli F, Leroy BP, ... ... High KA, et al. Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials. Ophthalmology. 126: 1273-1285. PMID 31443789 DOI: 10.1016/J.Ophtha.2019.06.017  0.331
2019 George LA, Sullivan SK, Rasko JEJ, Giermasz A, Samelson-Jones BJ, Ducore JM, Teitel JM, McGuinn CE, Runowski AR, Wright F, Anguela XM, High KA, Rybin D, Murphy JE, Rupon J. Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year Blood. 134: 3347-3347. DOI: 10.1182/Blood-2019-124091  0.387
2019 Drack AV, Bennett J, Russell S, High KA, Yu Z, Tillman A, Chung D, Reape KZ, Ciulla T, Maguire A. How long does gene therapy last? 4-year follow-up of phase 3 voretigene neparvovec trial in RPE65-associated LCA/inherited retinal disease Journal of Aapos. 23. DOI: 10.1016/J.Jaapos.2019.08.018  0.341
2018 Anguela XM, High KA. Entering the Modern Era of Gene Therapy. Annual Review of Medicine. PMID 30477394 DOI: 10.1146/Annurev-Med-012017-043332  0.372
2018 Dunbar CE, High KA, Joung JK, Kohn DB, Ozawa K, Sadelain M. Gene therapy comes of age. Science (New York, N.Y.). 359. PMID 29326244 DOI: 10.1126/Science.Aan4672  0.421
2018 Robinson M, George LA, Samelson-Jones BJ, Arruda VR, High KA, Carr ME, Tiefenbacher S. Activity of a FIX-Padua Transgene Product in Commonly Used FIX:C One-Stage and Chromogenic Assay Systems Following PF-06838435 (SPK-9001) Gene Delivery Blood. 132: 2198-2198. DOI: 10.1182/Blood-2018-99-119616  0.333
2018 High KA, George LA, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Samelson-Jones BJ, Evans M, Joseney-Antoine M, Macdougall A, Kadosh J, Runoski AR, Campbell-Baird C, Douglas K, Tompkins S, et al. A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds Blood. 132: 487-487. DOI: 10.1182/Blood-2018-99-115495  0.385
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  0.34
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  0.456
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  0.474
2017 George LA, Ragni MV, Samelson-Jones BJ, Cuker A, Runoski AR, Cole G, Wright F, Chen Y, Hui DJ, Wachtel K, Takefman D, Couto LB, Reape KZ, Carr ME, Anguela XM, ... High KA, et al. Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a Blood. 130: 604-604. DOI: 10.1182/Blood.V130.Suppl_1.604.604  0.469
2017 George LA, Sullivan SK, Giermasz A, Samelson-Jones BJ, Ducore JM, Teitel JM, Cuker A, Mackensen SV, Majumdar S, McGuinn CE, Runoski AR, Wright F, Dasen S, Barber K, Chen Y, ... ... High KA, et al. Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence Blood. 130: 601-601. DOI: 10.1182/Blood.V130.Suppl_1.601.601  0.42
2017 Hui DJ, Liu Y, Patel R, Chen Y, George LA, Sullivan SK, Giermasz A, Rasko J, Samelson-Jones BJ, Ducore JM, Dasen S, Carr ME, Anguela XM, High KA. Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response Blood. 130: 2056-2056. DOI: 10.1182/Blood.V130.Suppl_1.2056.2056  0.447
2016 Anguela XM, High KA. An edible switch for gene therapy. Nature Biotechnology. 34: 824-5. PMID 27504775 DOI: 10.1038/Nbt.3645  0.38
2016 Huang SS, High K, Toso R. Ocular Gene Therapy-The Future Is Now. Asia-Pacific Journal of Ophthalmology (Philadelphia, Pa.). 5: 227-8. PMID 27488063 DOI: 10.1097/Apo.0000000000000216  0.369
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  0.369
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  0.6
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  0.627
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  0.489
2016 George LA, Sullivan SK, Giermasz A, Ducore JM, Teitel JM, Cuker A, Sullivan LM, Majumdar S, McGuinn CE, Galvao AM, Luk AY, Wright JF, Chen Y, Hui DJ, Wachtel K, ... ... High KA, et al. Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression Blood. 128: 3-3. DOI: 10.1182/Blood.V128.22.3.3  0.448
2016 Leroy B, Maguire A, Russell S, Wellman J, Yu Z, Chung D, High K, Bennett J. Current gene therapy trials for inherited retinal disorders Acta Ophthalmologica. 94. DOI: 10.1111/J.1755-3768.2016.0160  0.37
2016 Hui DJ, Chen Y, Antrilli T, Liu Y, Corbau R, Russell SR, Maguire AM, Bennett J, High KA. 185. Safety Study by Validated Immunoassays in a Phase III Study of Subjects with Inherited Retinal Dystrophy Due to Mutations in the Gene Encoding Human Retinal Pigment Epithelium-Specific Protein 65 (RPE65) Injected with Adeno-Associated Viral Vectors Molecular Therapy. 24: S72-S73. DOI: 10.1016/S1525-0016(16)32994-X  0.416
2016 Anguela XM, Elkouby L, Toso R, DiPietro M, Davidson RJ, High KA, Sabatino DE. 52. Therapeutic Factor VIII Expression After AAV Delivery in Non-Human Primates Molecular Therapy. 24: S52. DOI: 10.1016/S1525-0016(16)32861-1  0.485
2016 Ignatova I, Falabella P, High KA, Couto LB. 31. AAV Gene Therapy for Choroideremia: Dose Determination Analyses Molecular Therapy. 24: S14. DOI: 10.1016/S1525-0016(16)32840-4  0.422
2016 Drack AV, Chung D, Russell S, Bennett J, Wellman J, Yu ZF, Maguire A, Stone EM, High K. Results of phase III clinical trial subretinal gene therapy for RPE65-mediated Leber congenital amaurosis (LCA) Journal of American Association For Pediatric Ophthalmology and Strabismus. 20: e4. DOI: 10.1016/J.Jaapos.2016.07.015  0.342
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  0.385
2015 High KA, Anguela XM. Adeno-associated viral vectors for the treatment of hemophilia. Human Molecular Genetics. PMID 26614390 DOI: 10.1093/Hmg/Ddv475  0.486
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  0.475
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  0.456
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  0.493
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  0.369
2015 Anguela XM, Sharma R, Doyon Y, Wechsler T, Paschon D, Davidson RJ, Zhou S, Gregory PD, Holmes MC, High KA. In Vivo Genome Editing in Neonatal Mouse Liver Preferentially Utilizes Homology Directed Repair Blood. 126: 4422-4422. DOI: 10.1182/Blood.V126.23.4422.4422  0.434
2015 Herzog RW, Hoffman BE, Zolotukhin I, High KA, Srivastava A, de Jong YP, Vercauteren K, Xiao J. AAV3 Capsid Is Superior for In Vivo Gene Transfer to Human Hepatocytes Compared to Serotypes 5 and 8 in a Mouse/Human Chimeric Model Blood. 126: 4418-4418. DOI: 10.1182/Blood.V126.23.4418.4418  0.624
2015 Anguela XM, Elkouby L, Toso R, DiPietro M, Davidson RJ, High KA, Sabatino DE. Adeno-Associated Viral Vector Delivery of Optimized Human Factor VIII Achieves Therapeutic Factor VIII Levels in Non-Human Primates Blood. 126: 199-199. DOI: 10.1182/Blood.V126.23.199.199  0.505
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  0.472
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  0.477
2014 High KA. Gene therapy for hemophilia: the clot thickens. Human Gene Therapy. 25: 915-22. PMID 25397928 DOI: 10.1089/Hum.2014.2541  0.311
2014 High K, Gregory PD, Gersbach C. CRISPR technology for gene therapy Nature Medicine. 20: 476-477. PMID 24804755 DOI: 10.1038/Nm.3566  0.338
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  0.655
2014 Elkouby L, Davidson RJ, Margaritis P, High KA, Sabatino DE. Improved Factor VIII Expression with the Introduction of a PACE-Furin Variant into Codon-Optimized Human Factor VIII Blood. 124: 3498-3498. DOI: 10.1182/Blood.V124.21.3498.3498  0.458
2014 Connolly CT, Faella A, Nichols TC, High KA, Arruda VR, Margaritis P. VKORc1 Is Under-Expressed in Skeletal Muscle of Humans, Dogs and Mice: Potential Implications for Ectopic Coagulation Factor Expression in Pre-Clinical and Therapeutic Applications Blood. 124: 1477-1477. DOI: 10.1182/Blood.V124.21.1477.1477  0.429
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  0.8
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  0.618
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  0.45
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  0.414
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  0.801
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  0.462
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  0.354
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  0.306
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  0.596
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  0.481
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  0.302
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  0.584
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  0.619
2013 Cousens LP, Najafian N, Mingozzi F, Elyaman W, Mazer B, Moise L, Messitt TJ, Su Y, Sayegh M, High K, Khoury SJ, Scott DW, De Groot AS. In vitro and in vivo studies of IgG-derived Treg epitopes (Tregitopes): a promising new tool for tolerance induction and treatment of autoimmunity. Journal of Clinical Immunology. 33: S43-9. PMID 22941509 DOI: 10.1007/S10875-012-9762-4  0.442
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  0.383
2013 Anguela XM, Sharma R, Doyon Y, Wechsler T, Paschon DE, Davidson RJ, Zhou S, Gregory PD, Holmes MC, Rebar EJ, High KA. ZFN Mediated Targeting Of Albumin “Safe Harbor” Results In Therapeutic Levels Of Human Factor VIII In a Mouse Model Of Hemophilia A Blood. 122: 720-720. DOI: 10.1182/Blood.V122.21.720.720  0.502
2013 Basner-Tschakarjan E, Mingozzi F, Chen Y, Nathwani A, Tuddenham E, Riddell A, Ng C, Rustagi P, Glader B, Kay M, Reiss UM, Nienhuis AW, Davidoff A, High KA. Cellular Immune Responses To Vector In a Gene Therapy Trial For Hemophilia B Using An AAV8 Self-Complementary Factor IX Vector Blood. 122: 717-717. DOI: 10.1182/Blood.V122.21.717.717  0.426
2013 Crudele JM, Finn JD, Martin NB, Siner JI, Chen Y, Zhou S, Niemeyer G, Lothrop CD, Mingozzi F, High KA, Arruda VR. Tolerance Induction To FIX Padua With AAV Liver Gene Transfer In Inhibitor-Prone Hemophilia B Dogs Blood. 122: 4203-4203. DOI: 10.1182/Blood.V122.21.4203.4203  0.483
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  0.466
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  0.509
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  0.466
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  0.63
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  0.329
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  0.506
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  0.813
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  0.625
2012 Davidoff A, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Chowdary P, Riddell A, Glader B, Rustagi P, Ng C, Kay M, Zhou J, Spence Y, Morton C, Allay J, ... ... High KA, et al. Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B Blood. 120: 752-752. DOI: 10.1182/Blood.V120.21.752.752  0.494
2012 Anguela XM, Sharma R, Doyon Y, Wong SY, Paschon DE, Li H, Haurigot V, Davidson RJ, Zhou S, Gregory PD, Holmes MC, Rebar E, High KA. In Vivo Genome Editing of Liver Albumin for Therapeutic Gene Expression: Rescue of Hemophilic Mice Via Integration of Factor 9 Blood. 120: 751-751. DOI: 10.1182/Blood.V120.21.751.751  0.67
2012 Crudele JM, Van Sluis GL, Margaritis P, Siner JI, Sliozberg M, Maurer J, Faella A, Zhou S, High KA, Esmon CT, Spek CA, Arruda VR. Insights Into the Mechanism of Zymogen Protein C Protection Against Cancer Progression Blood. 120: 3350-3350. DOI: 10.1182/Blood.V120.21.3350.3350  0.366
2012 Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, Hinderer CJ, Faella A, Howard C, Tai A, Podsakoff GM, Ragni MV, Zhou S, Basner-Tschakarjan E, Wright JF, ... High KA, et al. A Novel Strategy to Circumvent Pre-Existing Humoral Immunity to AAV Blood. 120: 2050-2050. DOI: 10.1182/Blood.V120.21.2050.2050  0.5
2012 Markusic DM, Martino AT, Mingozzi F, High KA, Herzog RW. In Vivo Model to Evaluate Loss of Liver-Derived Factor IX Expression Caused by AAV Capsid-Specific CD8+ T Cells Blood. 120: 2046-2046. DOI: 10.1182/Blood.V120.21.2046.2046  0.657
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  0.492
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  0.466
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  0.383
2011 Cheng L, Blazar B, High K, Porteus M. Zinc fingers hit off target. Nature Medicine. 17: 1192-3. PMID 21988995 DOI: 10.1038/Nm1011-1192  0.354
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  0.485
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  0.619
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  0.637
2011 Mingozzi F, High KA. Immune responses to AAV in clinical trials. Current Gene Therapy. 11: 321-30. PMID 21557723 DOI: 10.2174/156652311796150354  0.4
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  0.792
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  0.461
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  0.48
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  0.396
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  0.499
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  0.592
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  0.495
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  0.654
2011 Anguela XM, Sharma R, Li H, Haurigot V, Bhagwat A, Davidson RJ, Zhou S, Doyon Y, Gregory PD, Holmes MC, High KA. Robust Factor IX Expression Following ZFN-Mediated Genome Editing in An Adult Mouse Model of Hemophilia B Blood. 118: 668-668. DOI: 10.1182/Blood.V118.21.668.668  0.64
2011 Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh JH, Linch DC, Chowdary P, Griffioen A, Riddell A, Pie J, Harrington C, O’Beirne J, Smith K, Pasi J, Glader B, ... ... High KA, et al. Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B Blood. 118: 5-5. DOI: 10.1182/Blood.V118.21.5.5  0.485
2010 Wright JF, Wellman J, High KA. Manufacturing and regulatory strategies for clinical AAV2-hRPE65. Current Gene Therapy. 10: 341-9. PMID 20712582 DOI: 10.2174/156652310793180715  0.445
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  0.404
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  0.375
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  0.43
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  0.408
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  0.336
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  0.832
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  0.427
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  0.827
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  0.399
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  0.436
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: 10.1182/Blood.V114.22.695.695  0.502
2010 Li H, Haurigot V, Doyon Y, Li J, Bhagwat A, Wong S, Anguela X, Sharma R, Ivanciu L, Murphy S, Zhou S, Paschon D, Rebar E, Gregory PD, Holmes MC, ... High KA, et al. Phenotypic Correction of a Mouse Model of Hemophilia B by In Vivo Genetic Correction of the F9 Gene Blood. 116: LBA-5-LBA-5. DOI: 10.1182/Blood.V116.21.Lba-5.Lba-5  0.671
2010 Sluis GLV, Margaritis P, Sliozberg M, Mauer J, Faella A, Zhou S, High KA, Spek A, Arruda VR. Zymogen Protein C as a Novel Modulator of Cancer Progression In Murine Models Blood. 116: 718-718. DOI: 10.1182/Blood.V116.21.718.718  0.38
2010 Buchlis G, Mingozzi F, Soto PC, Pearce O, Hui DJ, Varki AP, High KA. Intrinsically Hyperactive and Hyperproliferative CD8+ T Cells In Cmah-/- Mice as a Model of Human Gene Transfer Responses. Blood. 116: 3773-3773. DOI: 10.1182/Blood.V116.21.3773.3773  0.375
2010 Hui DJ, Basner-Tschakarjan E, Pien GC, Martin WD, DeGroot AS, High KA, Mingozzi F. Peptide-Induced Antigen-Specific CD4+CD25+FoxP3+ T Cells Suppress Cytotoxicity T Cell Responses Directed Against the AAV Capsid. Blood. 116: 3769-3769. DOI: 10.1182/Blood.V116.21.3769.3769  0.369
2010 Finn JD, Favaro P, Wright JF, Mingozzi F, High KA, Arruda VR. Rabbit Anti-Thymocyte Globulin (rATG) Administrated Concomitantly with Liver Delivery of AAV2-hFIX Can Promote Inhibitor Formation In Rhesus Macaques. Blood. 116: 3765-3765. DOI: 10.1182/Blood.V116.21.3765.3765  0.512
2010 Nathwani A, Tuddenham E, Rosales C, McIntosh J, Riddell A, Rustagi P, Glader B, Kay M, Allay J, Coleman J, Sleep S, High KA, Mingozzi F, Gray JT, Reiss UM, et al. Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B Blood. 116: 248-248. DOI: 10.1182/Blood.V116.21.248.248  0.427
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  0.383
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  0.309
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  0.817
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  0.449
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  0.507
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  0.678
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  0.441
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 DOI: 10.1093/Ilar.50.2.144  0.459
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  0.432
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  0.505
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  0.587
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  0.347
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  0.634
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  0.466
2009 Finn JD, Simioni P, Iacobelli N, Zhou S, Nichols TC, High KA, Arruda VR. FIX-R338L (FIX Padua) as a Successful Alternative for the Treatment of Canine Severe Hemophilia B. Blood. 114: 694-694. DOI: 10.1182/Blood.V114.22.694.694  0.481
2009 Hui DJ, Pien GC, Basner-Tschakarjan E, Mingozzi F, Finn JD, Zhou S, Martin W, Groot ASD, High KA. Suppression of CTL Responses against AAV-Capsid Epitopes by Peptide-Induced Regulatory T Cells. Blood. 114: 377-377. DOI: 10.1182/Blood.V114.22.377.377  0.406
2009 Roy E, Faella A, Downey H, Ivanciu L, Zhou S, High KA, Margaritis P. Efficacy and Safety of Continuous Expression of An Improved Variant of FVIIa On Murine Hemostasis with or without Inhibitors to Human Factor IX. Blood. 114: 2466-2466. DOI: 10.1182/Blood.V114.22.2466.2466  0.468
2009 Li H, Malani N, Hamilton SR, Schlachterman A, Zhang JZ, Bussadori G, Edmonson SA, Mingozzi F, Wright JF, Arruda VR, Bushman FD, High KA. Assessment of Insertional Mutagenesis Risk Following AAV Vector-Mediated Factor IX Gene Transfer in Mice. Blood. 114: 2465-2465. DOI: 10.1182/Blood.V114.22.2465.2465  0.622
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  0.575
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  0.4
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  0.301
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  0.378
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  0.371
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  0.508
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  0.459
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  0.439
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  0.433
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  0.58
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  0.65
2008 Margaritis P, Roy E, Aljamali MN, Downey H, Zhou S, Merricks E, Dillow A, Nichols TC, High KA. Successful and Safe Treatment of Canine Hemophilia by Continuous Expression of Canine FVIIa: a Model for FVIII/FIX Gene-Based Bypassing Agents Blood. 112: lba-4-lba-4. DOI: 10.1182/Blood.V112.11.Lba-4.Lba-4  0.488
2008 Hui DJ, Mingozzi F, Kleefstra A, Meulenberg JM, Edmonson S, Morin D, Gaudet D, van Deventer S, High KA. Immunosuppression Modulates Immune Responses to AAV Capsid in Human Subjects Undergoing Intramuscular Gene Transfer for Lipoprotein Lipase Deficiency Blood. 112: 822-822. DOI: 10.1182/Blood.V112.11.822.822  0.485
2008 Liu Y, Margaritis P, Khazi F, Downey H, Kadauke S, Hasbrouck N, Sheedy J, Welch E, Weetall M, High KA. Nonsense Suppression Approaches in Treating Hemophilia Blood. 112: 512-512. DOI: 10.1182/Blood.V112.11.512.512  0.399
2008 Lin C, Kao C, Hong C, Ye P, Miao CH, Hamaguchi N, Wu H, Shi G, Yang Y, Yu I, Tao M, Fang C, Liu Y, High KA, Lin S. Engineered Factor IX with Augmented Clotting Activities in a Hemophilia B Mouse Model. Blood. 112: 2025-2025. DOI: 10.1182/Blood.V112.11.2025.2025  0.483
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  0.471
2007 Mingozzi F, High KA. Immune responses to AAV in clinical trials. Current Gene Therapy. 7: 316-24. PMID 17979678 DOI: 10.2174/156652307782151425  0.516
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  0.331
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  0.505
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  0.449
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  0.466
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  0.661
2007 Pien GC, Hasbrouck NC, Maus MV, Mingozzi F, High KA. Quantifying Capsid Peptide:MHC I Complexes Following Adeno-Associated Virus (AAV) Transduction Blood. 110: 3737-3737. DOI: 10.1182/Blood.V110.11.3737.3737  0.355
2007 Hauck B, Murphy S, Lui X, Zelenaia O, High KA, Wright JF. Trace Capsid DNA Impurities in AAV2 Vectors Are Not Transcribed and Are Unlikely To Contribute to Capsid Peptide-MHC Class I Complexes Blood. 110: 3736-3736. DOI: 10.1182/Blood.V110.11.3736.3736  0.458
2007 Roy E, Margaritis P, Downey HD, High KA. In Vitro and In Vivo Characterization of a Murine Model of Suppressed Intrinsic Pathway Using a FX Variant: FX Roma . Blood. 110: 2697-2697. DOI: 10.1182/Blood.V110.11.2697.2697  0.37
2007 Favaro P, Downey HD, Mingozzi F, Wright F, Hauck B, High KA, Arruda VR. Safety of Recombinant Adeno-Associated Viral Vectors in a Large Animal Model. Blood. 110: 2586-2586. DOI: 10.1182/Blood.V110.11.2586.2586  0.484
2007 Margaritis P, Roy E, Downey HD, Zhou S, Merricks E, Dillow A, Nichols TC, High KA. Gene Transfer of Canine FVIIa Results in Partial Correction of Canine Hemophilia: A Model for FVIII/FIX Gene-Based Bypassing Agents. Blood. 110: 195-195. DOI: 10.1182/Blood.V110.11.195.195  0.464
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  0.518
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  0.413
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  0.441
2006 Lillicrap D, Vandendriessche T, High K. Cellular and genetic therapies for haemophilia Haemophilia. 12: 36-41. PMID 16683995 DOI: 10.1111/J.1365-2516.2006.01259.X  0.473
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  0.657
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  0.464
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  0.487
2006 Khazi FR, Chu KC, High KA. Transmembrane Gla Protein 4 as a Novel Modulator of ERK2. Blood. 108: 544-544. DOI: 10.1182/Blood.V108.11.544.544  0.304
2006 Mingozzi F, Maus MV, Sabatino DE, Hui DJ, Rasko JE, Ragni MV, Manno CS, Ertl HC, High KA. AAV-2 Capsid-Specific CD8+ T Cells Limit the Duration of Gene Therapy in Humans and Cross-React with AAV-8 Capsid. Blood. 108: 455-455. DOI: 10.1182/Blood.V108.11.455.455  0.461
2006 Niemeyer GP, Herzog RW, Mount JD, Arruda V, Tillson M, Hathcock J, High KA, Lothrop CD. Six-Year Follow-Up of Inhibitor Prone Hemophilia B Dogs Treated with Muscle and Liver-Directed AAV2 Mediated Factor IX Gene Therapy. Blood. 108: 3282-3282. DOI: 10.1182/Blood.V108.11.3282.3282  0.631
2006 Margaritis P, Roy E, High KA. A Murine FVIIa Variant with Increased Tissue Factor-Independent Intrinsic Activity Corrects the Murine Hemophilia B Phenotype Following AAV Administration: Implications for Therapeutic AAV Vector Doses. Blood. 108: 3276-3276. DOI: 10.1182/Blood.V108.11.3276.3276  0.469
2006 Murphy SL, Hui DJ, Edmonson S, High KA. Conserved Amino Acid Sequences in Parvovirus B19 and Adeno-Associated Virus Stimulate Functionally Cross-Reactive CD8 T Cells. Blood. 108: 3269-3269. DOI: 10.1182/Blood.V108.11.3269.3269  0.438
2006 Hui DJ, Mingozzi F, Maus MV, Tigges MA, Pierce GF, High KA. A Novel Splenocyte Approach for Characterizing T Cell Responses to Adeno-Associated Virus in the Normal Population: Implications on Gene Transfer. Blood. 108: 3258-3258. DOI: 10.1182/Blood.V108.11.3258.3258  0.429
2006 Maus MV, Mingozzi F, Sabatino DE, Hui D, Ragni MV, High KA. 730. T Cell Responses to AAV Vector Capsid in Normal Donors and Subjects Who Have Undergone Liver-Directed AAV-Mediated Gene Transfer Molecular Therapy. 13: S282. DOI: 10.1016/J.Ymthe.2006.08.810  0.453
2006 Hauck B, Bajaj J, Joyce S, Qu G, Zelenaia OA, High KA, Wright JF. 508. Development of Optimized AAV2 Vector Biosynthesis and Purification Processes To Support High Capacity Production of Clinical Vectors of High Purity and Potency Molecular Therapy. 13: S196. DOI: 10.1016/J.Ymthe.2006.08.578  0.41
2006 Hauck B, Mingozzi F, Arruda V, High KA, Wright JF. 110. Investigation of Biochemical Factors That May Influence Immunogencity of AAV2 Vectors Molecular Therapy. 13: S45. DOI: 10.1016/J.Ymthe.2006.08.130  0.455
2006 Hui DJ, Mingozzi F, Dillow A, McCorquodale S, Nichols TC, Arruda VR, High KA. 1086. T Cell Responses to Canine Factor IX and AAV Capsid Antigens in Hemophilia B Dogs after Intravascular Gene Delivery to Skeletal Muscle Molecular Therapy. 13: S417. DOI: 10.1016/J.Ymthe.2006.08.1188  0.494
2006 Murphy SL, Sabatino D, Mingozzi F, Edmonson S, High K. 74. Cellular Immunity to Adeno-Associated Virus Capsid Attenuates Transgene Expression in the Liver Molecular Therapy. 13: S31-S32. DOI: 10.1016/J.Ymthe.2006.08.091  0.432
2006 Aljamali MN, Margaritis P, Arruda VR, Schlachterman A, Dunn D, Ware J, High KA. 66. Gene Transfer-Mediated Expression of Murine Factor VIIa Improves Clot Formation in a Mouse Model for Bernard-Soulier Syndrome Molecular Therapy. 13: S28. DOI: 10.1016/J.Ymthe.2006.08.082  0.42
2006 Toromanoff A, Cherel Y, Deschamps J, Arruda VR, High KA, Stedman HH, Haskins ME, Rolling F, Anegon I, Moullier P, Le Guiner C. 39. Evaluation of rAAV-1 vs rAAV-8 Vectors and Their Mode of Delivery in Nonhuman Primate Skeletal Muscle Molecular Therapy. 13: S16-S17. DOI: 10.1016/J.Ymthe.2006.08.052  0.36
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  0.419
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  0.37
2005 High K. Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients? Journal of Thrombosis and Haemostasis : Jth. 3: 1682-91. PMID 16102034 DOI: 10.1111/J.1538-7836.2005.01460.X  0.458
2005 High KA. Gene therapy: the moving finger. Nature. 435: 577-9. PMID 15931206 DOI: 10.1038/435577A  0.378
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  0.317
2005 High K. Anemia and gene therapy--a matter of control. The New England Journal of Medicine. 352: 1146-7. PMID 15784669 DOI: 10.1056/Nejmcibr044437  0.396
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  0.649
2005 Margaritis P, Tai SJ, Camire RM, Dunn D, High KA. Murine Model of Suppressed Intrinsic Pathway Using a FX Variant: FX Roma. Blood. 106: 728-728. DOI: 10.1182/Blood.V106.11.728.728  0.409
2005 Jiang H, Patarroyo-White S, Liu T, Yang B, Nagy D, He L, Scallan CD, Sommer J, Zhou S, Couto LB, High KA, Pierce GF. AAV8-Factor IX Hepatic Gene Transfer with Transient Immunosuppression Is Safe but Is Abrogated by Low Titers of Neutralizing Antibody in Rhesus Macaques. Blood. 106: 5532-5532. DOI: 10.1182/Blood.V106.11.5532.5532  0.491
2005 Mingozzi F, Maus MV, Sabatino DE, Hui D, Manno CS, Ragni MV, High KA. T Cell Responses to AAV Vector Capsid Limit the Duration of Transgene Expression in Humans after Liver-Directed Gene Therapy. Blood. 106: 3055-3055. DOI: 10.1182/Blood.V106.11.3055.3055  0.478
2005 Cao O, Armstrong E, Conti-Fine BM, High KA, Herzog RW. Suppression of Inhibitor Formation to F.IX in Gene Transfer through Immune Deviation Induced by Mucosal Peptide Administration. Blood. 106: 211-211. DOI: 10.1182/Blood.V106.11.211.211  0.628
2005 Aljamali MN, Margaritis P, Camire R, High KA. Lessons from Transgenic Mice Expressing Supra-Physiological Levels of Activated Murine Factor VII. Blood. 106: 1945-1945. DOI: 10.1182/Blood.V106.11.1945.1945  0.312
2005 Hui DJ, Mingozzi F, Sabatino DE, McCorquodale S, Dillow A, Nichols TC, Arruda VR, High KA. Characterization of the Immune Response to Canine Factor IX Following AAV-Mediated Intravascular Gene Delivery to Skeletal Muscle in Hemophilia B Dogs. Blood. 106: 1297-1297. DOI: 10.1182/Blood.V106.11.1297.1297  0.452
2005 Brooks DC, Khazi FR, High KA. 734. Association of DNA Polymerase |[mu]| with Recombinant Adeno-Associated Viral Vectors Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.274  0.359
2005 Sabatino D, Mingozzi F, Liu Y, Hui D, Tigges M, Pierce G, High K. 614. Frequency of T Cell Responses to AAV-2 Capsid in Peripheral Blood Mononuclear Cells of Normal Subjects Does Not Correlate with Anti-AAV2 Antibody Titers Molecular Therapy. 11: 238. DOI: 10.1016/J.Ymthe.2005.07.154  0.416
2005 Chen J, Hsu H, Schlachterman A, High KA, Mountz JD. 612. Determination of Specific Th1 and Th2 CD4 T Cell Responses in AAV2- and AAV8-Mediated Human Factor IX Gene Therapy in Mice Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.152  0.426
2005 Mingozzi F, Sabatino D, Nichols T, McCroquodale S, Arruda V, High K. 609. Long Term Persistence of Canine Factor IX-Specific Th1 Lymphocytes in Regional Lymph Nodes after AAV1-Mediated Intramuscular Gene Transfer in a Hemophilia B Dog Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.149  0.484
2005 Chen J, Hsu H, Schlachterman A, High KA, Mountz JD. 608. Cytotoxic T Lymphocyte Response to AAV2- and AAV8-Mediated Human Factor IX Gene Therapy in Mice Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.148  0.477
2005 Aljamali MN, Callan MB, Pollak ES, Giger U, Werner P, Griot-Wenk M, High KA. 606. Molecular Characterization of Hereditary Factor VII Deficiency in Beagles Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.146  0.344
2005 Hauck B, Xu R, Xie J, Ding Q, Wu W, Sipler M, Wang H, High K, Xiao W. 605. Efficient AAV 1 and AAV2 Hybrid Vector for Gene Therapy of Hemophilia Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.145  0.475
2005 Arruda V, Stedman H, Jian H, Pierce G, Schuettrumpf J, Freguia CF, Baila S, Gopal K, Nichols T, High K. 603. Correction of Hemophilia B Phenotype by Novel Method of Regional Intravenous Delivery of AAV Vector to Skeletal Muscle of Hemophilia B Dogs Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.143  0.46
2005 Hsieh M, Liu Y, Schlachterman A, High KA. 397. AAV-Mediated Gene Transfer to Endothelial Cells Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.400  0.455
2005 Schlachterman A, Margaritis P, Aljamali M, Liu J, Arruda V, High K. 84. Assessing Hemostatic Efficacy of Continuous Expression of Factor VIIa after AAV-Mediated, Liver-Directed Gene Transfer in Hemophilia A and B Mice Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.062  0.423
2005 Aljamali MN, Margaritis P, Camire RM, High KA. 39. Effects of Long-Term Expression of Activated Murine FVII in Normal and Hemophilic Mice Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.038  0.343
2005 Khazi FR, Schlachterman A, High KA. 14. rAAV2 Integration Junctions Isolated without In Vivo Selection Bias Show a Preference to 5|[prime]| Regions of Open Reading Frames Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.017  0.343
2005 Hsieh M, Khazi FR, Schlachterman A, Liu Y, High KA. 13. Persistence of AAV Capsid in Transduced Cells Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.016  0.461
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  0.662
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  0.631
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  0.477
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  0.467
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  0.684
2004 Schlachterman A, Liu J, Liu Y, High K, Arruda V. In Vivo Evidence of Modulation of the Hemophilia Phenotype by the Factor V Leiden. Blood. 104: 693-693. DOI: 10.1182/Blood.V104.11.693.693  0.357
2004 Schuettrumpf J, Zou J, Tai SJ, Schlachterman A, Tian K, Edmonson S, Liu J, Andrade-Gordon P, High K, Arruda V. A Novel Role of Coagulation Proteases on Viral-Based Gene Transfer Efficacy. Blood. 104: 691-691. DOI: 10.1182/Blood.V104.11.691.691  0.491
2004 Aljamali MN, Margaritis P, Schlachterman A, High KA. Generation of Transgenic Mice Expressing High Levels of Activated Murine Coagulation Factor VII. Blood. 104: 5253-5253. DOI: 10.1182/Blood.V104.11.5253.5253  0.434
2004 Cao O, Armstrong E, Conti-Fine BM, High KA, Herzog RW. Prevention of Gene Transfer-Induced Inhibitor Formation by Nasal Administration of Human F.IX T Cell Epitope in a Murine Model of Hemophilia B. Blood. 104: 414-414. DOI: 10.1182/Blood.V104.11.414.414  0.658
2004 High K, Tigges M, Manno C, Sabatino D, Arruda V, Herzog R, Rustagi P, Rasko J, Sommer J, Jaworski K, Ragni M, Glader B, Lessard R, Luk A, Couto L, et al. Human Immune Responses to AAV-2 Capsid May Limit Duration of Expression in Liver-Directed Gene Transfer in Humans with Hemophilia B. Blood. 104: 413-413. DOI: 10.1182/Blood.V104.11.413.413  0.619
2004 Sabatino DE, Mingozzi F, Chen H, Colosi P, Ertl HC, High KA. Identification of the AAV2 Capsid CD8+ T Cell Epitope in C57BL/6 Mice. Blood. 104: 3188-3188. DOI: 10.1182/Blood.V104.11.3188.3188  0.39
2004 Schlachterman A, Liu J, Aljamali M, Arruda VR, High KA. Assessing Hemostatic Efficacy of Continuous Expression of Factor VIIa Following Administration of AAV Vectors into Hemophilia B Mice. Blood. 104: 3184-3184. DOI: 10.1182/Blood.V104.11.3184.3184  0.419
2004 Liu Y, Zhu H, Schlachterman A, Chang H, Camire RM, High KA. A Novel Deletion in the Fviii B-Domain That Reduces Transgene Size While Preserving FVIII Activity. Blood. 104: 3182-3182. DOI: 10.1182/Blood.V104.11.3182.3182  0.525
2004 Arruda VR, Stedman HH, Schuettrumpf J, Jiang H, Pierce G, Nichols TC, High KA. A Novel Method of Regional Intravenous Delivery of AAV Vector to Skeletal Muscle Results in Correction of Canine Hemophilia B Phenotype. Blood. 104: 3179-3179. DOI: 10.1182/Blood.V104.11.3179.3179  0.468
2004 Tai SJ, Hudson P, Freguia CF, High KA. Expression of Blood Coagulation Factor X Is Not Liver-Restricted. Blood. 104: 1939-1939. DOI: 10.1182/Blood.V104.11.1939.1939  0.378
2004 High KA. Gutted adenoviral vectors in hemophilia A Blood. 103: 751-752. DOI: 10.1182/Blood-2003-11-3996  0.387
2004 Zhang H, Hsu H, Yang P, Wu Q, Yu S, Wang J, Schlachterman A, High KA, Mountz JD. 1047. BXD Recombinant Inbred Mice Represent a Novel Immune Response Model to AAV-F.IX Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.992  0.399
2004 High K, Manno C, Sabatino D, Hutchison S, Dake M, Razavi M, Kaye R, Aruda V, Herzog R, Rustagi P, Rasko J, Hoots K, Blatt P, Sommer J, Ragni M, et al. 1002. Immune Responses to AAV and to Factor IX in a Phase I Study of AAV-Mediated, Liver-Directed Gene Transfer for Hemophilia B Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.940  0.643
2004 Zaoui K, Schmidt M, Sabatino DE, Khazi FR, Glimm H, Sun J, High KA, Kalle Cv. 345. Sensitive Molecular Detection of Various Stable Episomal Concatemeric and Integrated Forms of rAAV2 after Long-Term Expression In Vivo Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.295  0.461
2004 Khazi FR, Edmondson AC, High KA. 307. A Mechanism for AZT Mediated Enhancement of Transgene Expression Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.249  0.438
2004 Liu Y, Zhu H, Schlachterman A, Chang H, Carime RM, High KA. 104. A novel Deletion in the FVIII B-Domain That Reduces Transgene Size While Preserving FVIII Activity Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.041  0.456
2004 Arruda V, Scallan C, Jiang H, Couto L, Herzog RW, Nichols T, Pierce G, High KA. 103. Comparison of the Efficacy on Gene Transfer by AAV Vectors Delivered by Distinct Routes of Administration to the Liver of Hemophilia B Dogs Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.040  0.624
2004 Sabatino DE, Mingozzi F, Brooks DC, Schlachterman A, Edmonson S, Couto L, Colosi P, Chen H, Ertl HCJ, High KA. 37. Duration of Immunologically Detectable AAV-2 Capsid in an Animal Model Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.05.060  0.478
2004 Aljamali MN, Margaritis P, Arruda VR, Schlachterman A, Camire RM, High KA. 32. Efficacy and Safety of Mouse Coagulation Factor VIIa Gene Transfer in Hemophilia B Mice Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.05.055  0.464
2003 Kohn DB, Sadelain M, Dunbar C, Bodine D, Kiem HP, Candotti F, Tisdale J, Riviére I, Blau CA, Richard RE, Sorrentino B, Nolta J, Malech H, Brenner M, Cornetta K, ... ... High K, et al. American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Molecular Therapy : the Journal of the American Society of Gene Therapy. 8: 180-7. PMID 12907140 DOI: 10.1016/S1525-0016(03)00212-0  0.434
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  0.45
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  0.314
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  0.678
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  0.476
2003 High K. AAV-mediated gene transfer for hemophilia. Genetics in Medicine : Official Journal of the American College of Medical Genetics. 4: 56S-61S. PMID 12544490 DOI: 10.1097/00125817-200211001-00012  0.422
2003 High K. AAV-mediated gene transfer for hemophilia. Genetics in Medicine : Official Journal of the American College of Medical Genetics. 4: 56S-61S. PMID 12544490 DOI: 10.1097/00125817-200211001-00012  0.422
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  0.682
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  0.526
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 DOI: 10.1128/Jvi.76.20.10497-10502.2002  0.659
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  0.613
2002 High K. Gene-based approaches to the treatment of hemophilia. Annals of the New York Academy of Sciences. 961: 63-4. PMID 12081866 DOI: 10.1111/J.1749-6632.2002.Tb03050.X  0.321
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 DOI: 10.1182/Blood.V99.8.2670  0.656
2002 High KA. In vivo gene transfer moves one step closer to success Blood. 100: 1523-1524. DOI: 10.1182/Blood-2002-07-2008  0.445
2002 High KA. Gene Therapy for Hemophilia The Biomedical & Life Sciences Collection. 385-410. DOI: 10.1002/0471223956.Ch15  0.371
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  0.596
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 DOI: 10.1007/978-1-4615-1277-6_4  0.391
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  0.651
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  0.649
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  0.608
2001 High KA. Gene therapy: A 2001 perspective Haemophilia. 7: 23-27. PMID 11240615 DOI: 10.1046/J.1365-2516.2001.00098.X  0.426
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  0.317
2001 High KA. Gene transfer as an approach to treating hemophilia Circulation Research. 88: 137-144. PMID 11157664 DOI: 10.1161/01.Res.88.2.137  0.426
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  0.601
2000 Hedner U, Ginsburg D, Lusher JM, High KA. Congenital Hemorrhagic Disorders: New Insights into the Pathophysiology and Treatment of Hemophilia. Hematology. American Society of Hematology. Education Program. 241-265. PMID 11701545 DOI: 10.1182/Asheducation-2000.1.241  0.388
2000 High KA. Gene therapy in haematology and oncology Lancet. 356. PMID 11191528 DOI: 10.1016/S0140-6736(00)91994-9  0.368
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  0.303
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 DOI: 10.1055/S-0037-1614021  0.544
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  0.65
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 DOI: 10.1182/Blood.V95.8.2536.008K26_2536_2542  0.645
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  0.67
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 DOI: 10.1055/S-0037-1615877  0.683
1999 Kay MA, High K. Gene therapy for the hemophilias Proceedings of the National Academy of Sciences of the United States of America. 96: 9973-9975. PMID 10468539 DOI: 10.1073/Pnas.96.18.9973  0.347
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  0.676
1998 Herzog RW, High KA. Problems and prospects in gene therapy for hemophilia Current Opinion in Hematology. 5: 321-326. PMID 9776210 DOI: 10.1097/00062752-199809000-00003  0.674
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 DOI: 10.1182/Blood.V92.5.1639  0.351
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 DOI: 10.1182/Blood.V91.12.4600.412K22_4600_4607  0.635
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 DOI: 10.1182/Blood.V91.3.784.784_784_790  0.413
1998 Hagstrom JN, Herzog R, Arruda V, Chu K, Fields P, Tai SJ, Couto LB, Kurtzman GJ, High KA, Manno CS. #513 Muscle targeted adeno-associated viral vector mediated gene therapy for hemophilia B Journal of Pediatric Hematology/Oncology. 20: 371. DOI: 10.1097/00043426-199807000-00035  0.317
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  0.45
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  0.655
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  0.306
1997 Fisher KJ, Jooss K, Alston J, Yang Y, Haecker SE, High K, Pathak R, Raper SE, Wilson JM. Recombinant adeno-associated virus for muscle directed gene therapy. Nature Medicine. 3: 306-12. PMID 9055858 DOI: 10.1038/Nm0397-306  0.484
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 DOI: 10.1182/Blood.V89.1.176  0.325
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  0.306
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  0.314
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  0.463
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 DOI: 10.1074/Jbc.271.3.1738  0.33
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  0.301
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 DOI: 10.1007/978-1-4613-0331-2_6  0.317
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  0.432
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 DOI: 10.1182/Blood.V83.12.3524.Bloodjournal83123524  0.331
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 DOI: 10.1089/Hum.1994.5.3-313  0.391
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  0.337
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  0.311
1992 Larson PJ, High KA. Biology of inherited coagulopathies: Factor IX Hematology/Oncology Clinics of North America. 6: 999-1009. PMID 1356960 DOI: 10.1016/S0889-8588(18)30289-2  0.325
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 DOI: 10.1172/Jci115484  0.369
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  0.338
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 DOI: 10.1182/Blood.V75.5.1097.Bloodjournal7551097  0.33
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 DOI: 10.1182/Blood.V74.5.1486.Bloodjournal7451486  0.306
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 DOI: 10.1182/Blood.V74.1.207.Bloodjournal741207  0.384
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 DOI: 10.1182/Blood.V73.6.1540.Bloodjournal7361540  0.307
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  0.319
1989 Huang M, Kasper C, Roberts H, Stafford D, High K. Molecular defect in factor IXHilo, a hemophilia Bm variant: Arg----Gln at the carboxyterminal cleavage site of the activation peptide Blood. 73: 718-721. DOI: 10.1182/Blood.V73.3.718.Bloodjournal733718  0.302
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 DOI: 10.1172/Jci112814  0.604
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  0.526
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