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Katherine High

Research Profile

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David Callejas; Tura Ferre; Felix Garcia; Iris Grifoll; Virginia Haurigot; Katherine A High; Ricardo Lage; Christopher J Mann; Federico Mingozzi; Jordi Montané; Sergio Muñoz; Carles Roca; Jesús Ruberte; Rafael Ruiz-de Gopegui; Shangzhen Zhou; Anna Andaluz; Eduard Ayuso; Fatima Bosch
Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.
Elizabeth M Parzych; Gregory M Podsakoff; Te-Lang Wu; Xiangfan Yin; Katherine A High; Matthew H Levine; Hua Li; Qin Liu; Hildegund C J Ertl
Effects of Immunosuppression on Circulating Adeno-Associated Virus Capsid-Specific T cells in Humans.
Federico Mingozzi; P P Tak; R M Thurlings; Y Chen; S C Edmonson; Katherine A High; S Zhou; M J Vervoordeldonk
Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue.

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All Publications

2013: David Callejas; Tura Ferre; Felix Garcia; Iris Grifoll; Virginia Haurigot; Katherine A High; Ricardo Lage; Christopher J Mann; Federico Mingozzi; Jordi Montané; Sergio Muñoz; Carles Roca; Jesús Ruberte; Rafael Ruiz-de Gopegui; Shangzhen Zhou; Anna Andaluz; Eduard Ayuso; Fatima Bosch
Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.
Diabetes 2013;62(5):1718-29.
2013: Elizabeth M Parzych; Gregory M Podsakoff; Te-Lang Wu; Xiangfan Yin; Katherine A High; Matthew H Levine; Hua Li; Qin Liu; Hildegund C J Ertl
Effects of Immunosuppression on Circulating Adeno-Associated Virus Capsid-Specific T cells in Humans.
Human gene therapy 2013;24(4):431-42.
2013: Federico Mingozzi; P P Tak; R M Thurlings; Y Chen; S C Edmonson; Katherine A High; S Zhou; M J Vervoordeldonk
Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue.
Gene therapy 2013;20(4):417-24.
2013: Ashley T Martino; Federico Mingozzi; David A Ostrov; Arun Srivastava; Cox Terhorst; Shangzhen Zhou; Etiena Basner-Tschakarjan; Hildegund C J Ertl; Jonathan D Finn; Katherine A High; Christian Hinderer; David M Markusic; Roland W Herzog
Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.
Blood 2013;121(12):2224-33.
2012: Jonathan D Finn; Katherine A High; Elizabeth P Merricks; Timothy C Nichols; Paolo Simioni; Nikolaos Svoronos; Shangshen Zhou; Dwight A Bellenger; Valder R Arruda
The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.
Blood 2012;120(23):4521-3.
2012: Katherine A High
The gene therapy journey for hemophilia: are we there yet?
Blood 2012;120(23):4482-7.
2012: David A Suhy; Laurence Whiteley; J Fraser Wright; Olga Zelenaia; Shangzhen Zhou; Kevin Brady; Andrew D Burdick; Hubert Denise; Bernd Hauck; Kyle Hayes; Katherine A High; Shih-Chu Kao; Alexander Kolykhalov; Helen Lavender; Tin Mao; Sterghios A Moschos; Sara H Renison; Peter Roelvink; Curt Scribner; Bernard Souberbielle; Romu Corbau
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 2012;20(9):1737-49.
2012: Federico Mingozzi; Samuel L Murphy; Sandra D Price; Alex Tai; J Fraser Wright; Shangzhen Zhou; Yifeng Chen; Robert E Donahue; Cynthia E Dunbar; Shyrie Edmonson; Mark E Metzger; Katherine A High
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 2012;20(7):1410-6.
2012: George Buchlis; Alan W Flake; Sarah M Hawk; Federico Mingozzi; Gregory M Podsakoff; Antonetta Radu; Katherine A High
Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.
Blood 2012;119(13):3038-41.
2012: Jean Bennett; Jeannette L Bennicelli; Yifeng Chen; Daniel C Chung; Laura L Cyckowski; Katherine A High; Manzar Ashtari; Alberto Auricchio; Kathleen A Marshall; Sarah McCague; Federico Mingozzi; Eric A Pierce; Kenneth S Shindler; Francesca Simonelli; Junwei Sun; Jennifer Wellman; J Fraser Wright; Gui-Shuang Ying; Xiaosong Zhu; Albert M Maguire
AAV2 gene therapy readministration in three adults with congenital blindness.
Science translational medicine 2012;4(120):120ra15.
2012: Douglas B Cines; Katherine A High; Morris A Blajchman; James B Bussel
Clinical trial opportunities in hemostasis and thrombosis: NHBLI State-of-the-science symposium.
American journal of hematology 2012;87(2):235-8.
2012: Brad E Hoffman; Cox Terhorst; Hildegund C J Ertl; Katherine A High; Roland W Herzog
Gene therapy research at the frontiers of viral immunology.
Frontiers in microbiology 2012;3():182.
2011: Mallory R Scola; Hongtu Zhu; Melissa C Caughey; Katherine A High; Paris Margaritis; Elizabeth P Merricks; Timothy C Nichols; Robin A Raymer; Caterina M Gallippi
ARFI ultrasound monitoring of hemorrhage and hemostasis in vivo in canine von Willebrand disease and hemophilia.
Ultrasound in medicine & biology 2011;37(12):2126-32.
2011: Hua Li; Shih-Wen Lin; Larissa H Haut; Katherine A High; Bei Jia; Marcio O Lasaro; Hildegund C J Ertl
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 2011;19(11):2021-30.
2011: Federico Mingozzi; Katherine A High
Immune responses to AAV in clinical trials.
Current gene therapy 2011;11(4):321-30.
2011: Lawrence T Bish; George Buchlis; Guangping Gao; Jeffrey Gazzara; Katherine A High; Daniel Hui; Caryn Reynolds; Gretchen E Singletary; Meg M Sleeper; James M Wilson; Elanor Withnall; H Lee Sweeney
Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines.
Human gene therapy 2011;22(8):969-77.
2011: Federico Mingozzi; Katherine A High
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
Nature reviews. Genetics 2011;12(5):341-55.
2011: Paris Margaritis; Elise Roy; Armida Faella; Harre D Downey; Lacramioara Ivanciu; Giulia Pavani; Shangzhen Zhou; Ralph M Bunte; Katherine A High
Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo.
Blood 2011;117(15):3974-82.
2011: Jingmin Zhou; Xiao Yang; J Fraser Wright; Katherine A High; Linda Couto; Guang Qu
PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9.
Journal of virological methods 2011;173(1):99-107.
2011: Hojun Li; Nirav Malani; Shari R Hamilton; Alexander Schlachterman; Giulio Bussadori; Shyrie Edmonson; Rachel Shah; Valder R Arruda; Federico Mingozzi; J Fraser Wright; Frederic D Bushman; Katherine A High
Assessing the potential for AAV vector genotoxicity in a murine model.
Blood 2011;117(12):3311-9.
2011: Hua Li; Steven Tuyishime; Te-Lang Wu; Wynetta Giles-Davis; Dongming Zhou; Weidong Xiao; Katherine A High; Hildegund C J Ertl
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 2011;19(3):536-46.
2010: Linda B Couto; Katherine A High
Viral vector-mediated RNA interference.
Current opinion in pharmacology 2010;10(5):534-42.
2010: Virginia Haurigot; Federico Mingozzi; George Buchlis; Daniel J Hui; Yifeng Chen; Etiena Basner-Tschakarjan; Valder R Arruda; Antoneta Radu; Helen G Franck; J Fraser Wright; Shangzhen Zhou; Hansell H Stedman; Dwight A Bellinger; Timothy C Nichols; Katherine A High
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 2010;18(7):1318-29.
2010: Valder R Arruda; Hansell H Stedman; Virginia Haurigot; George Buchlis; Stefano Baila; Patricia Favaro; Yifeng Chen; Helen G Franck; Shangzhen Zhou; J Fraser Wright; Linda B Couto; Haiyan Jiang; Glenn F Pierce; Dwight A Bellinger; Federico Mingozzi; Timothy C Nichols; Katherine A High
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.
Blood 2010;115(23):4678-88.
2010: Paris Margaritis; Katherine A High
Gene therapy in haemophilia--going for cure?
Haemophilia : the official journal of the World Federation of Hemophilia 2010;16 Suppl 3():24-8.
2010: Timothy C Nichols; Robin A Raymer; H W G Franck; Elizabeth P Merricks; Dwight A Bellinger; N DeFriess; P Margaritis; Valder R Arruda; M A Kay; Katherine A High
Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.
Haemophilia : the official journal of the World Federation of Hemophilia 2010;16 Suppl 3():19-23.
2010: Eduard Ayuso; Federico Mingozzi; J Montane; X Leon; X M Anguela; V Haurigot; Shyrie Edmonson; L Africa; S Zhou; Katherine A High; Fatima Bosch; J F Wright
High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency.
Gene therapy 2010;17(4):503-10.
2010: Defne Amado; Federico Mingozzi; Daniel Hui; Jeannette L Bennicelli; Zhangyong Wei; Yifeng Chen; Erin Bote; Rebecca L Grant; Jeffrey A Golden; Kristina Narfstrom; Nasreen A Syed; Stephen E Orlin; Katherine A High; Albert M Maguire; Jean Bennett
Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness.
Science translational medicine 2010;2(21):21ra16.
2010: Jonathan D Finn; Daniel Hui; Harre D Downey; Danielle Dunn; Gary C Pien; Federico Mingozzi; Shangzhen Zhou; Katherine A High
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 2010;18(1):135-42.
2009: Ou Cao; Brad E Hoffman; Babak Moghimi; Sushrusha Nayak; Mario Cooper; Shangzhen Zhou; Hildegund C J Ertl; Katherine A High; Roland W Herzog
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 2009;17(10):1733-42.
2009: Federico Mingozzi; Janneke J Meulenberg; Daniel J Hui; Etiena Basner-Tschakarjan; Nicole C Hasbrouck; Shyrie Edmonson; Natalie A Hutnick; Michael R Betts; John J Kastelein; Erik S Stroes; Katherine A High
AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells.
Blood 2009;114(10):2077-86.
2009: Hua Li; Shih-Wen Lin; Wynetta Giles-Davis; Yan Li; Dongming Zhou; Zhi Q Xiang; Katherine A High; Hildegund C J Ertl
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 2009;17(7):1215-24.
2009: Gary C Pien; Etiena Basner-Tschakarjan; Daniel J Hui; Ashley N Mentlik; Jonathan D Finn; Nicole C Hasbrouck; Shangzhen Zhou; Samuel L Murphy; Marcela V Maus; Federico Mingozzi; Jordan S Orange; Katherine A High
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors.
The Journal of clinical investigation 2009;119(6):1688-95.
2009: Patricia Favaro; Harre D Downey; J Shangzhen Zhou; J Fraser Wright; Bernd Hauck; Federico Mingozzi; Katherine A High; Valder R Arruda
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 2009;17(6):1022-30.
2009: Paris Margaritis; Elise Roy; Majed N Aljamali; Harre D Downey; Urs Giger; Shangzhen Zhou; Elizabeth Merricks; Aaron Dillow; Mirella Ezban; Timothy C Nichols; Katherine A High
Successful treatment of canine hemophilia by continuous expression of canine FVIIa.
Blood 2009;113(16):3682-9.
2009: Sapna Gupta; Jirko Kühnisch; Aladdin Mustafa; Sarka Lhotak; Alexander Schlachterman; Michael J Slifker; Andres Klein-Szanto; Katherine A High; Richard C Austin; warren kruger
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 2009;23(3):883-93.
2009: Glenn P Niemeyer; Roland W Herzog; Jane D Mount; Valder R Arruda; D Michael Tillson; John Hathcock; Frederik W van Ginkel; Katherine A High; Clinton D Lothrop
Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy.
Blood 2009;113(4):797-806.
2009: Timothy C Nichols; Aaron M Dillow; Helen W G Franck; Elizabeth P Merricks; Robin A Raymer; Dwight A Bellinger; Valder R Arruda; Katherine A High
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 2009;50(2):144-67.
2009: Samuel L Murphy; Hojun Li; Federico Mingozzi; Denise E Sabatino; Daniel J Hui; Shyrie Edmonson; Katherine A High
Diverse IgG subclass responses to adeno-associated virus infection and vector administration.
Journal of medical virology 2009;81(1):65-74.
2009: Bernd Hauck; Samuel L Murphy; Peter H Smith; Guang Qu; Xingge Liu; Olga Zelenaia; Federico Mingozzi; Jurg Sommer; Katherine A High; J Fraser Wright
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 2009;17(1):144-52.
2008: Samuel L Murphy; Anand Bhagwat; Shyrie Edmonson; Shangzhen Zhou; Katherine A High
High-throughput screening and biophysical interrogation of hepatotropic AAV.
Molecular therapy : the journal of the American Society of Gene Therapy 2008;16(12):1960-7.
2008: Alice Toromanoff; Yan Chérel; Mickaël Guilbaud; Magalie Penaud-Budloo; Richard O Snyder; Mark E Haskins; Jack-Yves Deschamps; Lydie Guigand; Guillaume Podevin; Valder R Arruda; Katherine A High; Hansell H Stedman; Fabienne Rolling; Ignacio Anegon; Philippe Moullier; Caroline Le Guiner
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 2008;16(7):1291-9.
2008: N C Hasbrouck; Katherine A High
AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects.
Gene therapy 2008;15(11):870-5.
2008: Majed N Aljamali; Paris Margaritis; Alexander Schlachterman; Shing Jen Tai; Elise Roy; Ralph Bunte; Rodney M Camire; Katherine A High
Long-term expression of murine activated factor VII is safe, but elevated levels cause premature mortality.
The Journal of clinical investigation 2008;118(5):1825-34.
2008: Samuel L Murphy; Katherine A High
Gene therapy for haemophilia.
British journal of haematology 2008;140(5):479-87.
2008: Jeannette Bennicelli; J Fraser Wright; Andras Komaromy; Jonathan B Jacobs; Bernd Hauck; Olga Zelenaia; Federico Mingozzi; Daniel Hui; Daniel Chung; Tonia Rex; Zhangyong Wei; Guang Qu; Shangzhen Zhou; Caroline J Zeiss; Valder R Arruda; Gregory M Acland; Louis Dell'Osso; Katherine A High; Albert M Maguire; Jean Bennett
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 2008;16(3):458-65.
2008: Shing Jen Tai; Roland W Herzog; Paris Margaritis; Valder R Arruda; K Chu; J A Golden; P A Labosky; Katherine A High
A viable mouse model of factor X deficiency provides evidence for maternal transfer of factor X.
Journal of thrombosis and haemostasis : JTH 2008;6(2):339-45.
2007: Federico Mingozzi; Katherine A High
Immune responses to AAV in clinical trials.
Current gene therapy 2007;7(5):316-24.
2007: Federico Mingozzi; Nicole C Hasbrouck; Etiena Basner-Tschakarjan; Shyrie Edmonson; Daniel J Hui; Denise E Sabatino; Shangzhen Zhou; J Fraser Wright; Haiyan Jiang; Glenn F Pierce; Valder R Arruda; Katherine A High
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver.
Blood 2007;110(7):2334-41.
2007: Chunmei Yang; Jinong Feng; Wenjia Song; Jicheng Wang; Becky Tsai; Yunwu Zhang; William A Scaringe; Kathleen A Hill; Paris Margaritis; Katherine A High; Steve S Sommer
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 2007;104(39):15394-9.
2007: Denise E Sabatino; Tippi C Mackenzie; William H Peranteau; Shyrie Edmonson; Cesare Campagnoli; Yi-Lin Liu; Alan W Flake; Katherine A High
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 2007;15(9):1677-85.
2007: Federico Mingozzi; Marcela V Maus; Daniel J Hui; Denise E Sabatino; Samuel L Murphy; John Rasko; Margaret V Ragni; Catherine S Manno; Jurg M Sommer; Haiyan Jiang; Glenn F Pierce; Hildegund C J Ertl; Katherine A High
CD8(+) T-cell responses to adeno-associated virus capsid in humans.
Nature medicine 2007;13(4):419-22.
2007: Hua Li; Samuel L Murphy; Wynetta Giles-Davis; Shyrie Edmonson; Zhiquan Xiang; Yan Li; Marcio O Lasaro; Katherine A High; Hildegund C J Ertl
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 2007;15(4):792-800.
2007: Guang Qu; Jennifer Bahr-Davidson; Joseph Prado; Alex Tai; Floro Cataniag; Jennifer McDonnell; Jingmin Zhou; Bernd Hauck; Jac Luna; Jurg M Sommer; Peter Smith; Shangzhen Zhou; Peter Colosi; Katherine A High; Glenn F Pierce; J Fraser Wright
Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography.
Journal of virological methods 2007;140(1-2):183-92.
2006: Mary Beth Callan; M N Aljamali; Paris Margaritis; ME Griot-Wenk; Eleanor S Pollak; P Werner; Urs Giger; Katherine A High
A novel missense mutation responsible for factor VII deficiency in research Beagle colonies.
Journal of thrombosis and haemostasis : JTH 2006;4(12):2616-22.
2006: Haiyan Jiang; Linda B Couto; Susannah Patarroyo-White; Tongyao Liu; Dea Nagy; Joseph Vargas; Shangzhen Zhou; Ciaran D Scallan; Jurg M Sommer; Sharmila Vijay; Federico Mingozzi; Katherine A High; Glenn F Pierce
Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy.
Blood 2006;108(10):3321-8.
2006: Haiyan Jiang; Glenn F Pierce; Margareth C Ozelo; Erich V de Paula; Joseph Vargas; Peter Smith; Jurg M Sommer; Alvin Luk; Catherine S Manno; Katherine A High; Valder R Arruda
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 2006;14(3):452-5.
2006: Ou Cao; Elina Armstrong; Alexander Schlachterman; Lixin Wang; David K Okita; Bianca M Conti-Fine; Katherine A High; Roland W Herzog
Immune deviation by mucosal antigen administration suppresses gene-transfer-induced inhibitor formation to factor IX.
Blood 2006;108(2):480-6.
2006: Katherine A High
The leak stops here: platelets as delivery vehicles for coagulation factors.
The Journal of clinical investigation 2006;116(7):1840-2.
2006: Paris Margaritis; Katherine A High
Advances in gene therapy using factor VIIa in hemophilia.
Seminars in hematology 2006;43(1 Suppl 1):S101-4.
2005: Denise E Sabatino; Federico Mingozzi; Daniel J Hui; Haifeng Chen; Peter Colosi; Hildegund C J Ertl; Katherine A High
Identification of mouse AAV capsid-specific CD8+ T cell epitopes.
Molecular therapy : the journal of the American Society of Gene Therapy 2005;12(6):1023-33.
2005: Huang-Ge Zhang; Katherine A High; Qi Wu; Pingar Yang; Alexander Schlachterman; Shaohua Yu; Nengjun Yi; Hui-Chen Hsu; John D Mountz
Genetic analysis of the antibody response to AAV2 and factor IX.
Molecular therapy : the journal of the American Society of Gene Therapy 2005;11(6):866-74.
2005: Valder R Arruda; Hansell H Stedman; Timothy C Nichols; Mark E Haskins; Matthew Nicholson; Roland W Herzog; Linda B Couto; Katherine A High
Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model.
Blood 2005;105(9):3458-64.
2004: Denise E Sabatino; Elina Armstrong; Shyrie Edmonson; Yi-Lin Liu; Marc Pleimes; Joerg Schuettrumpf; Julie Fitzgerald; Roland W Herzog; Valder R Arruda; Katherine A High
Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene.
Blood 2004;104(9):2767-74.
2004: Yi-Lin Liu; Federico Mingozzi; Sol Rodriguez-Colon; Sasha Joseph; Eric Dobrzynski; Takashi Suzuki; Katherine A High; Roland W Herzog
Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector.
Human gene therapy 2004;15(8):783-92.
2004: Herbert C Whinna; E B Lesesky; Dougald M Monroe; Katherine A High; Peter J Larson; Frank Church
Role of the gamma-carboxyglutamic acid domain of activated factor X in the presence of calcium during inhibition by antithrombin-heparin.
Journal of thrombosis and haemostasis : JTH 2004;2(7):1127-34.
2004: Katherine A High
Clinical gene transfer studies for hemophilia B.
Seminars in thrombosis and hemostasis 2004;30(2):257-67.
2004: Paris Margaritis; Valder R Arruda; Majed Aljamali; Rodney M Camire; Alexander Schlachterman; Katherine A High
Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII.
The Journal of clinical investigation 2004;113(7):1025-31.
2004: Valder R Arruda; Joerg Schuettrumpf; Roland W Herzog; Timothy C Nichols; Nancy Robinson; Yasmin Lotfi; Federico Mingozzi; Weidong Xiao; Linda B Couto; Katherine A High
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 2004;103(1):85-92.
2003: Federico Mingozzi; Yi-Lin Liu; Eric Dobrzynski; Antje Kaufhold; Jian Hua Liu; YuQin Wang; Valder R Arruda; Katherine A High; Roland W Herzog
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer.
The Journal of clinical investigation 2003;111(9):1347-56.
2003: Catherine S Manno; Amy J Chew; Sylvia Hutchison; Peter J Larson; Roland W Herzog; Valder R Arruda; Shing Jen Tai; Margaret V Ragni; Arthur Thompson; Margareth C Ozelo; Linda B Couto; Debra G B Leonard; Frederick A Johnson; Alan McClelland; Ciaran D Scallan; Erik D Skarsgard; Alan W Flake; Mark A Kay; Katherine A High; Bertil Glader
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.
Blood 2003;101(8):2963-72.
2003: John Gerecitano; Clarissa Mathias; Rosemarie Mick; Kathleen M Duffy; Selina M Luger; Edward A Stadtmauer; Stephen J Schuster; Donald Tsai; Sunita D Nasta; Jesse Berlin; Deborah K Phillips; Katherine A High; David Porter
Homocysteine and prothrombin fragment 1+2 levels in patients with veno-occlusive disease after stem cell transplantation.
Journal of hematotherapy & stem cell research 2003;12(2):215-23.
2003: Raffaella Toso; francesco bernardi; Theresa Tidd; Mirko Pinotti; Rodney M Camire; Giovanna Marchetti; Katherine A High; Eleanor S Pollak
Factor VII mutant V154G models a zymogen-like form of factor VIIa.
The Biochemical journal 2003;369(Pt 3):563-71.
2002: Katherine A High
Adeno-associated virus-mediated gene transfer for hemophilia B.
International journal of hematology 2002;76(4):310-8.
2002: Federico Mingozzi; Jörg Schüttrumpf; Valder R Arruda; Yuhong Liu; Yi-Lin Liu; Katherine A High; Weidong Xiao; Roland W Herzog
Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector.
Journal of virology 2002;76(20):10497-502.
2002: Roland W Herzog; Paul A Fields; Valder R Arruda; Jeff O Brubaker; Elina Armstrong; Darryl McClintock; Dwight A Bellinger; Linda B Couto; Timothy C Nichols; Katherine A High
Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy.
Human gene therapy 2002;13(11):1281-91.
2002: Raffaella Toso; Mirko Pinotti; Katherine A High; Eleanor S Pollak; francesco bernardi
A frequent human coagulation Factor VII mutation (A294V, c152) in loop 140s affects the interaction with activators, tissue factor and substrates.
The Biochemical journal 2002;363(Pt 2):411-6.
2002: Jane D Mount; Roland W Herzog; D Michael Tillson; Susan A Goodman; Nancy Robinson; Mark L McCleland; Dwight A Bellinger; Timothy C Nichols; Valder R Arruda; Clinton D Lothrop; Katherine A High
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy.
Blood 2002;99(8):2670-6.
2001: Katherine A High
AAV-mediated gene transfer for hemophilia.
Annals of the New York Academy of Sciences 2001;953():64-74.
2001: Valder R Arruda; Paul A Fields; R Milner; Luanne M Wainwright; M P De Miguel; P J Donovan; Roland W Herzog; Timothy C Nichols; Jaclyn A Biegel; M Razavi; M Dake; D Huff; Alan W Flake; Linda B Couto; Mark A Kay; Katherine A High
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 2001;4(6):586-92.
2001: Paul A Fields; Valder R Arruda; Elina Armstrong; K Chu; Federico Mingozzi; J Nathan Hagstrom; Roland W Herzog; Katherine A High
Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9.
Molecular therapy : the journal of the American Society of Gene Therapy 2001;4(3):201-10.
2001: Roland W Herzog; Jane D Mount; Valder R Arruda; Katherine A High; Clinton D Lothrop
Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation.
Molecular therapy : the journal of the American Society of Gene Therapy 2001;4(3):192-200.
2001: Paul A Fields; Elina Armstrong; J Nathan Hagstrom; Valder R Arruda; M L Murphy; Jay P Farrell; Katherine A High; Roland W Herzog
Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice.
Gene therapy 2001;8(5):354-61.
2001: H L Hung; E S Pollak; Rama Kudaravalli; Valder R Arruda; K Chu; Katherine A High
Regulation of human coagulation factor X gene expression by GATA-4 and the Sp family of transcription factors.
Blood 2001;97(4):946-51.
2001: Peter J Larson; Katherine A High
Gene therapy for hemophilia B: AAV-mediated transfer of the gene for coagulation factor IX to human muscle.
Advances in experimental medicine and biology 2001;489():45-57.
2001: Valder R Arruda; J Nathan Hagstrom; J Deitch; Terry D Heiman-Patterson; Rodney M Camire; K Chu; Paul A Fields; Roland W Herzog; Linda B Couto; Peter J Larson; Katherine A High
Posttranslational modifications of recombinant myotube-synthesized human factor IX.
Blood 2001;97(1):130-8.
2000: Rodney M Camire; Peter J Larson; Darrel W Stafford; Katherine A High
Enhanced gamma-carboxylation of recombinant factor X using a chimeric construct containing the prothrombin propeptide.
Biochemistry 2000;39(46):14322-9.
2000: Roland W Herzog; V R Arruda; T H Fisher; Marjorie S Read; Timothy C Nichols; Katherine A High
Absence of circulating factor IX antigen in hemophilia B dogs of the UNC-Chapel Hill colony.
Thrombosis and haemostasis 2000;84(2):352-4.
2000: J Nathan Hagstrom; Linda B Couto; Ciaran D Scallan; M Burton; Mark L McCleland; Paul A Fields; Valder R Arruda; Roland W Herzog; Katherine A High
Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter.
Blood 2000;95(8):2536-42.
2000: Paul A Fields; D W Kowalczyk; Valder R Arruda; Elina Armstrong; Mark L McCleland; J Nathan Hagstrom; K John Pasi; Hildegund C J Ertl; Roland W Herzog; Katherine A High
Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX.
Molecular therapy : the journal of the American Society of Gene Therapy 2000;1(3):225-35.
2000: Mark A Kay; Catherine S Manno; Margaret V Ragni; Peter J Larson; Linda B Couto; Alan McClelland; B Glader; A J Chew; Shing Jen Tai; Roland W Herzog; Valder R Arruda; F Johnson; Ciaran D Scallan; Erik D Skarsgard; Alan W Flake; Katherine A High
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.
Nature genetics 2000;24(3):257-61.
1999: Roland W Herzog; Katherine A High
Adeno-associated virus-mediated gene transfer of factor IX for treatment of hemophilia B by gene therapy.
Thrombosis and haemostasis 1999;82(2):540-6.
1999: Roland W Herzog; E Y Yang; Linda B Couto; J Nathan Hagstrom; D Elwell; Paul A Fields; M Burton; Dwight A Bellinger; Marjorie S Read; Kenneth Brinkhous; Greg M Podsakoff; Timothy C Nichols; GJ Kurtzman; Katherine A High
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector.
Nature medicine 1999;5(1):56-63.
1998: J Nathan Hagstrom; J Walter; R Bluebond-Langner; J C Amatniek; Catherine S Manno; Katherine A High
Prevalence of the factor V leiden mutation in children and neonates with thromboembolic disease.
The Journal of pediatrics 1998;133(6):777-81.
1998: Roland W Herzog; Katherine A High
Problems and prospects in gene therapy for hemophilia.
Current opinion in hematology 1998;5(5):321-6.
1998: JA Carew; E S Pollak; Katherine A High; Kenneth A Bauer
Severe factor VII deficiency due to a mutation disrupting an Sp1 binding site in the factor VII promoter.
Blood 1998;92(5):1639-45.
1998: Hiroyuki Nakai; Roland W Herzog; J Nathan Hagstrom; J Walter; SH Kung; E Y Yang; Shing Jen Tai; Y Iwaki; GJ Kurtzman; K J Fisher; Peter Colosi; Linda B Couto; Katherine A High
Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver.
Blood 1998;91(12):4600-7.
1998: Peter J Larson; Rodney M Camire; D Wong; N C Fasano; Dougald M Monroe; Paula B Tracy; Katherine A High
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 1998;37(14):5029-38.
1998: SH Kung; J Nathan Hagstrom; D Cass; Shing Jen Tai; Hui-Feng Lin; Darrel W Stafford; Katherine A High
Human factor IX corrects the bleeding diathesis of mice with hemophilia B.
Blood 1998;91(3):784-90.
1997: Roland W Herzog; J Nathan Hagstrom; SH Kung; Shing Jen Tai; James M Wilson; K J Fisher; Katherine A High
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 1997;94(11):5804-9.
1997: AA Arbini; E S Pollak; J K Bayleran; Katherine A High; Kenneth A Bauer
Severe factor VII deficiency due to a mutation disrupting a hepatocyte nuclear factor 4 binding site in the factor VII promoter.
Blood 1997;89(1):176-82.
1996: S Sridhara; S Chaing; Katherine A High; Morris A Blajchman; B J Clarke
Activation of a recombinant human factor VII structural analogue alters its affinity of binding to tissue factor.
American journal of hematology 1996;53(2):66-71.
1996: K Chu; SM Wu; T Stanley; Darrel W Stafford; Katherine A High
A mutation in the propeptide of Factor IX leads to warfarin sensitivity by a novel mechanism.
The Journal of clinical investigation 1996;98(7):1619-25.
1996: J Walter; Q You; J Nathan Hagstrom; M Sands; Katherine A High
Successful expression of human factor IX following repeat administration of adenoviral vector in mice.
Proceedings of the National Academy of Sciences of the United States of America 1996;93(7):3056-61.
1996: Peter J Larson; S A Stanfield-Oakley; W J VanDusen; C K Kasper; Kenneth J Smith; Dougald M Monroe; Katherine A High
Structural integrity of the gamma-carboxyglutamic acid domain of human blood coagulation factor IXa Is required for its binding to cofactor VIIIa.
The Journal of biological chemistry 1996;271(7):3869-76.
1996: H L Hung; Katherine A High
Liver-enriched transcription factor HNF-4 and ubiquitous factor NF-Y are critical for expression of blood coagulation factor X.
The Journal of biological chemistry 1996;271(4):2323-31.
1996: E S Pollak; H L Hung; W Godin; G C Overton; Katherine A High
Functional characterization of the human factor VII 5'-flanking region.
The Journal of biological chemistry 1996;271(3):1738-47.
1994: S Chaing; B Clarke; S Sridhara; K Chu; P Friedman; W VanDusen; Harold R Roberts; Morris A Blajchman; Dougald M Monroe; Katherine A High
Severe factor VII deficiency caused by mutations abolishing the cleavage site for activation and altering binding to tissue factor.
Blood 1994;83(12):3524-35.
1994: S H Chaing; A Wallmark; E Berntorp; Katherine A High
A NlaIII polymorphism within the human factor VII gene.
Human genetics 1994;93(6):722-3.
1994: J N Lozier; A R Thompson; P C Hu; M Read; Kenneth Brinkhous; Katherine A High; D T Curiel
Efficient transfection of primary cells in a canine hemophilia B model using adenovirus-polylysine-DNA complexes.
Human gene therapy 1994;5(3):313-22.
1993: Francesco Giannelli; Peter M Green; Katherine A High; S Sommer; M C Poon; M Ludwig; Rainer Schwaab; P H Reitsma; M Goossens; A Yoshioka
Haemophilia B: database of point mutations and short additions and deletions--fourth edition, 1993.
Nucleic acids research 1993;21(13):3075-87.
1993: S Sridhara; B J Clarke; Frederick A Ofosu; Katherine A High; Morris A Blajchman
The direct binding of human factor VII in plasma to recombinant human tissue factor.
Thrombosis research 1993;70(4):307-16.
1993: Marco Racchi; H H Watzke; Katherine A High; Mark Lively
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.
The Journal of biological chemistry 1993;268(8):5735-40.
1992: Peter J Larson; Katherine A High
Biology of inherited coagulopathies: factor IX.
Hematology/oncology clinics of North America 1992;6(5):999-1009.
1992: M N Huang; H L Hung; S A Stanfield-Oakley; Katherine A High
Characterization of the human blood coagulation factor X promoter.
The Journal of biological chemistry 1992;267(22):15440-6.
1992: Francesco Giannelli; Peter M Green; Katherine A High; S Sommer; D P Lillicrap; M Ludwig; K Olek; P H Reitsma; M Goossens; A Yoshioka
Haemophilia B: database of point mutations and short additions and deletions--third edition, 1992.
Nucleic acids research 1992;20 Suppl():2027-63.
1991: H H Watzke; A Wallmark; nobuko hamaguchi; Patricia J Giardina; D W Stafford; Katherine A High
Factor XSanto Domingo. Evidence that the severe clinical phenotype arises from a mutation blocking secretion.
The Journal of clinical investigation 1991;88(5):1685-9.
1991: A Wallmark; V L Rose; C Ho; Katherine A High
A NlaIV polymorphism within the human factor X gene.
Nucleic acids research 1991;19(14):4022.
1991: Francesco Giannelli; Peter M Green; Katherine A High; S Sommer; D P Lillicrap; M Ludwig; K Olek; P H Reitsma; M Goossens; A Yoshioka
Haemophilia B: database of point mutations and short additions and deletions--second edition.
Nucleic acids research 1991;19 Suppl():2193-219.
1990: M N Huang; Katherine A High
Efficient subcloning of DNA fragments amplified by crude oligonucleotides.
BioTechniques 1990;9(6):710-1.
1990: Francesco Giannelli; Peter M Green; Katherine A High; J N Lozier; D P Lillicrap; M Ludwig; K Olek; P H Reitsma; M Goossens; A Yoshioka
Haemophilia B: database of point mutations and short additions and deletions.
Nucleic acids research 1990;18(14):4053-9.
1990: H H Watzke; K Lechner; Harold R Roberts; Sakamuri V Reddy; D J Welsch; P Friedman; G Mahr; Pudur Jagadeeswaran; Dougald M Monroe; Katherine A High
Molecular defect (Gla+14----Lys) and its functional consequences in a hereditary factor X deficiency (factor X "Vorarlberg").
The Journal of biological chemistry 1990;265(20):11982-9.
1990: J N Lozier; Dougald M Monroe; S Stanfield-Oakley; S W Lin; Kenneth J Smith; Harold R Roberts; Katherine A High
Factor IX New London: substitution of proline for glutamine at position 50 causes severe hemophilia B.
Blood 1990;75(5):1097-104.
1990: J N Lozier; Katherine A High
Molecular basis of hemophilia.
Hematologic pathology 1990;4(1):1-26.
1989: James P Evans; Kenneth Brinkhous; G D Brayer; Howard Reisner; Katherine A High
Canine hemophilia B resulting from a point mutation with unusual consequences.
Proceedings of the National Academy of Sciences of the United States of America 1989;86(24):10095-9.
1989: Sakamuri V Reddy; ZQ Zhou; K J Rao; J P Scott; H Watzke; Katherine A High; Pudur Jagadeeswaran
Molecular characterization of human factor XSan Antonio.
Blood 1989;74(5):1486-90.
1989: James P Evans; H H Watzke; J L Ware; D W Stafford; Katherine A High
Molecular cloning of a cDNA encoding canine factor IX.
Blood 1989;74(1):207-12.
1989: Dougald M Monroe; D M McCord; M N Huang; Katherine A High; R L Lundblad; C K Kasper; Harold R Roberts
Functional consequences of an arginine180 to glutamine mutation in factor IX Hilo.
Blood 1989;73(6):1540-4.
1989: M N Huang; C K Kasper; Harold R Roberts; D W Stafford; Katherine A High
Molecular defect in factor IXHilo, a hemophilia Bm variant: Arg----Gln at the carboxyterminal cleavage site of the activation peptide.
Blood 1989;73(3):718-21.
1988: B G Macik; Don A Gabriel; G C White; Katherine A High; Harold R Roberts
The use of high-dose intravenous gamma-globulin in acquired von Willebrand syndrome.
Archives of pathology & laboratory medicine 1988;112(2):143-6.