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Mark Kay

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TOP 3
Jiamiao Lu; Feijie Zhang; Mark A Kay
A Mini-intronic Plasmid (MIP): A Novel Robust Transgene Expression Vector In Vivo and In Vitro.
Leszek Lisowski; Kirk Chu; Menashe Elazar; Jeffrey S Glenn; Mark A Kay
The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA.
Lia E Gracey Maniar; Zhi-Ying Chen; Andrew Z Fire; Jiamiao Lu; Jay M Maniar; Mark A Kay
Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level.

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All Publications

2013: Jiamiao Lu; Feijie Zhang; Mark A Kay
A Mini-intronic Plasmid (MIP): A Novel Robust Transgene Expression Vector In Vivo and In Vitro.
Molecular therapy : the journal of the American Society of Gene Therapy 2013;21(5):954-63.
2013: Leszek Lisowski; Kirk Chu; Menashe Elazar; Jeffrey S Glenn; Mark A Kay
The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA.
Nucleic acids research 2013;41(6):3688-98.
2013: Lia E Gracey Maniar; Zhi-Ying Chen; Andrew Z Fire; Jiamiao Lu; Jay M Maniar; Mark A Kay
Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level.
Molecular therapy : the journal of the American Society of Gene Therapy 2013;21(1):131-8.
2012: Shuo Gu; Yong Huang; Lan Jin; Paul N Valdmanis; Feijie Zhang; Yue Zhang; Mark A Kay
The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo.
Cell 2012;151(4):900-11.
2012: Paul N Valdmanis; Leszek Lisowski; Mark A Kay
rAAV-mediated tumorigenesis: still unresolved after an AAV assault.
Molecular therapy : the journal of the American Society of Gene Therapy 2012;20(11):2014-7.
2012: Jiamiao Lu; Siqun Xu; Feijie Zhang; Andrew Z Fire; Mark A Kay
The extragenic spacer length between the 5' and 3' ends of the transgene expression cassette affects transgene silencing from plasmid-based vectors.
Molecular therapy : the journal of the American Society of Gene Therapy 2012;20(11):2111-9.
2012: Leszek Lisowski; Zhongya Wang; Feijie Zhang; Yue Zhang; Markus Grompe; Ashley Lau; Mark A Kay
Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression.
Molecular therapy : the journal of the American Society of Gene Therapy 2012;20(10):1912-23.
2012: Shuo Gu; Yong Huang; L Jin; Feijie Zhang; Mark A Kay
Slicing-independent RISC activation requires the argonaute PAZ domain.
Current biology : CB 2012;22(16):1536-42.
2011: Mei Huang; Fangjun Jia; Mark A Kay; Divya Nag; Patricia Nguyen; Robert C Robbins; Li Wang; Patricia E de Almeida; Amato J Giaccia; Yongquan Gong; Shijun Hu; Joseph C Wu
Double knockdown of prolyl hydroxylase and factor-inhibiting hypoxia-inducible factor with nonviral minicircle gene therapy enhances stem cell mobilization and angiogenesis after myocardial infarction.
Circulation 2011;124(11 Suppl):S46-54.
2011: Shuo Gu; L Jin; Feijie Zhang; Yong Huang; Dirk Grimm; John J Rossi; Mark A Kay
Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes.
Proceedings of the National Academy of Sciences of the United States of America 2011;108(22):9208-13.
2011: Mark A Kay
State-of-the-art gene-based therapies: the road ahead.
Nature reviews. Genetics 2011;12(5):316-28.
2011: Mark J Osborn; Ron T McElmurry; Christopher J Lees; Anthony P DeFeo; Zhi-Ying Chen; Mark A Kay; Luigi Naldini; Gordon Freeman; Jakub Tolar; Bruce R Blazar
Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I.
Molecular therapy : the journal of the American Society of Gene Therapy 2011;19(3):450-60.
2011: Kazim H Narsinh; Fangjun Jia; Robert C Robbins; Mark A Kay; Michael T Longaker; Joseph C Wu
Generation of adult human induced pluripotent stem cells using nonviral minicircle DNA vectors.
Nature protocols 2011;6(1):78-88.
2010: Mark A Kay; Cheng-Yi He; Zhi-Ying Chen
A robust system for production of minicircle DNA vectors.
Nature biotechnology 2010;28(12):1287-9.
2010: Fangjun Jia; Kitchener D Wilson; Ning Sun; Deepak M Gupta; Mei Huang; Zongjin Li; Nicholas J Panetta; Zhi Ying Chen; Robert C Robbins; Mark A Kay; Michael T Longaker; Joseph C Wu
A nonviral minicircle vector for deriving human iPS cells.
Nature methods 2010;7(3):197-9.
2010: Dirk Haussecker; Mark A Kay
miR-122 continues to blaze the trail for microRNA therapeutics.
Molecular therapy : the journal of the American Society of Gene Therapy 2010;18(2):240-2.
2010: Lia E Gracey; Zhi-Ying Chen; Jay M Maniar; Anton Valouev; Arend Sidow; Mark A Kay; Andrew Fire
An in vitro-identified high-affinity nucleosome-positioning signal is capable of transiently positioning a nucleosome in vivo.
Epigenetics & chromatin 2010;3(1):13.
2010: Shuo Gu; Mark A Kay
How do miRNAs mediate translational repression?
Silence 2010;1(1):11.
2010: Shelly Beer; David I Bellovin; Joyce S Lee; Kimberly Komatsubara; Lora S Wang; Huishan Koh; Kathleen Börner; Theresa A Storm; Corrine R Davis; Mark A Kay; Dean W Felsher; Dirk Grimm
Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice.
Molecular therapy : the journal of the American Society of Gene Therapy 2010;18(1):161-70.
2009: Mei Huang; ZhiYing Chen; Shijun Hu; Fangjun Jia; Zongjin Li; Grant Hoyt; Robert C Robbins; Mark A Kay; Joseph C Wu
Novel minicircle vector for gene therapy in murine myocardial infarction.
Circulation 2009;120(11 Suppl):S230-7.
2008: Jeffery C Giering; Dirk Grimm; Theresa A Storm; Mark A Kay
Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic.
Molecular therapy : the journal of the American Society of Gene Therapy 2008;16(9):1630-6.
2008: Holger Doege; Dirk Grimm; Alaric Falcon; Bernice Tsang; Theresa A Storm; Hui Xu; Angelica M Ortegon; Melissa Kazantzis; Mark A Kay; Andreas Stahl
Silencing of hepatic fatty acid transporter protein 5 in vivo reverses diet-induced non-alcoholic fatty liver disease and improves hyperglycemia.
The Journal of biological chemistry 2008;283(32):22186-92.
2008: Xichen Zhang; Michael W Epperly; Mark A Kay; Zhi-Ying Chen; Tracy Dixon; Darcy Franicola; Benjamin A Greenberger; Paavani Komanduri; Joel S Greenberger
Radioprotection in vitro and in vivo by minicircle plasmid carrying the human manganese superoxide dismutase transgene.
Human gene therapy 2008;19(8):820-6.
2008: Dirk Haussecker; Dan Cao; Yong Huang; Poornima Parameswaran; Andrew Fire; Mark A Kay
Capped small RNAs and MOV10 in human hepatitis delta virus replication.
Nature structural & molecular biology 2008;15(7):714-21.
2008: Dirk Grimm; Joyce S Lee; Lora Wang; Tushar J Desai; Bassel Akache; Theresa A Storm; Mark A Kay
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses.
Journal of virology 2008;82(12):5887-911.
2008: Anton P McCaffrey; Paul Fawcett; Hiroyuki Nakai; Ramona L McCaffrey; Anja Ehrhardt; Thu-Thao T Pham; Kusum Pandey; Hui Xu; Sally Feuss; Theresa A Storm; Mark A Kay
The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver.
Molecular therapy : the journal of the American Society of Gene Therapy 2008;16(5):931-41.
2008: Zhi-Ying Chen; Efren Riu; Cheng-Yi He; Hui Xu; Mark A Kay
Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation.
Molecular therapy : the journal of the American Society of Gene Therapy 2008;16(3):548-56.
2008: Konrad L Streetz; Regis Doyonnas; Dirk Grimm; D Denison Jenkins; Sally Fuess; Scott Perryman; Joseph Lin; Christian Trautwein; Judith A Shizuru; Helen Blau; Karl G Sylvester; Mark A Kay
Hepatic parenchymal replacement in mice by transplanted allogeneic hepatocytes is facilitated by bone marrow transplantation and mediated by CD4 cells.
Hepatology (Baltimore, Md.) 2008;47(2):706-18.
2007: Dirk Grimm; Mark A Kay
Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?
The Journal of clinical investigation 2007;117(12):3633-41.
2007: Brian Moldt; Stephen R Yant; Peter Refsing Andersen; Mark A Kay; Jacob Giehm Mikkelsen
Cis-acting gene regulatory activities in the terminal regions of sleeping beauty DNA transposon-based vectors.
Human gene therapy 2007;18(12):1193-204.
2007: Brian S Garrison; Stephen R Yant; Jacob Giehm Mikkelsen; Mark A Kay
Postintegrative gene silencing within the Sleeping Beauty transposition system.
Molecular and cellular biology 2007;27(24):8824-33.
2007: Xuan Shen; Theresa A Storm; Mark A Kay
Characterization of the relationship of AAV capsid domain swapping to liver transduction efficiency.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(11):1955-62.
2007: Mark A Kay
AAV vectors and tumorigenicity.
Nature biotechnology 2007;25(10):1111-3.
2007: Daniel M Paskowitz; Kenneth P Greenberg; Douglas Yasumura; Dirk Grimm; Haidong Yang; Jacque L Duncan; Mark A Kay; Matthew M LaVail; John G Flannery; Douglas Vollrath
Rapid and stable knockdown of an endogenous gene in retinal pigment epithelium.
Human gene therapy 2007;18(10):871-80.
2007: Katsuya Inagaki; Congrong Ma; Theresa A Storm; Mark A Kay; Hiroyuki Nakai
The role of DNA-PKcs and artemis in opening viral DNA hairpin termini in various tissues in mice.
Journal of virology 2007;81(20):11304-21.
2007: Katsuya Inagaki; Susanna M Lewis; Xiaolin Wu; Congrong Ma; David J Munroe; Sally Fuess; Theresa A Storm; Mark A Kay; Hiroyuki Nakai
DNA palindromes with a modest arm length of greater, similar 20 base pairs are a significant target for recombinant adeno-associated virus vector integration in the liver, muscles, and heart in mice.
Journal of virology 2007;81(20):11290-303.
2007: Aaron D Tward; Kirk D Jones; Stephen Yant; Siu Tim Cheung; Sheung-Tat Fan; Xin Chen; Mark A Kay; Rong Wang; John M Bishop
Distinct pathways of genomic progression to benign and malignant tumors of the liver.
Proceedings of the National Academy of Sciences of the United States of America 2007;104(37):14771-6.
2007: Hisaya Azuma; Nicole Paulk; Aarati Ranade; Craig Dorrell; Muhsen Al-Dhalimy; Ewa Ellis; Stephen Strom; Mark A Kay; Milton Finegold; Markus Grompe
Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice.
Nature biotechnology 2007;25(8):903-10.
2007: Efren Riu; Zhi-Ying Chen; Hui Xu; Cheng-Yi He; Mark A Kay
Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(7):1348-55.
2007: Hatem Zayed; Lily Xia; Anton Yerich; Stephen R Yant; Mark A Kay; M Puttaraju; Gerard J McGarrity; David L. Wiest; R Scott McIvor; Jakub Tolar; Bruce R Blazar
Correction of DNA protein kinase deficiency by spliceosome-mediated RNA trans-splicing and sleeping beauty transposon delivery.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(7):1273-9.
2007: Dirk Grimm; Mark A Kay
Combinatorial RNAi: a winning strategy for the race against evolving targets?
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(5):878-88.
2007: Bassel Akache; Dirk Grimm; Xuan Shen; Sally Fuess; Stephen R Yant; Dariya S Glazer; Julie Park; Mark A Kay
A two-hybrid screen identifies cathepsins B and L as uncoating factors for adeno-associated virus 2 and 8.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(2):330-9.
2007: Alfred Y Wang; Anja Ehrhardt; Hui Xu; Mark A Kay
Adenovirus transduction is required for the correction of diabetes using Pdx-1 or Neurogenin-3 in the liver.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(2):255-63.
2007: Jakub Tolar; Alma J Nauta; Mark J Osborn; Angela Panoskaltsis-Mortari; Ron T McElmurry; Scott Bell; Lily Xia; Ning Zhou; Megan Riddle; Tania M Schroeder; Jennifer J Westendorf; R Scott McIvor; Pancras Hogendoorn; Karoly Szuhai; Leann Oseth; Betsy Hirsch; Stephen R Yant; Mark A Kay; Alexandra Peister; Darwin J Prockop; Willem E Fibbe; Bruce R Blazar
Sarcoma derived from cultured mesenchymal stem cells.
Stem cells (Dayton, Ohio) 2007;25(2):371-9.
2007: Stephen R Yant; Yong Huang; Bassel Akache; Mark A Kay
Site-directed transposon integration in human cells.
Nucleic acids research 2007;35(7):e50.
2007: Anja Ehrhardt; Stephen R Yant; Jeffery C Giering; Hui Xu; Jeffrey A Engler; Mark A Kay
Somatic integration from an adenoviral hybrid vector into a hot spot in mouse liver results in persistent transgene expression levels in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2007;15(1):146-56.
2006: Anja Ehrhardt; Jeffrey A Engler; Hui Xu; Athena M Cherry; Mark A Kay
Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration.
Human gene therapy 2006;17(11):1077-94.
2006: Bassel Akache; Dirk Grimm; Kusum Pandey; Stephen R Yant; Hui Xu; Mark A Kay
The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9.
Journal of virology 2006;80(19):9831-6.
2006: Katsuya Inagaki; Sally Fuess; Theresa A Storm; Gregory A Gibson; Charles F McTiernan; Mark A Kay; Hiroyuki Nakai
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8.
Molecular therapy : the journal of the American Society of Gene Therapy 2006;14(1):45-53.
2006: Dirk Grimm; Konrad L Streetz; Catherine L Jopling; Theresa A Storm; Kusum Pandey; Corrine R Davis; Patricia L Marion; Felix H Salazar; Mark A Kay
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways.
Nature 2006;441(7092):537-41.
2006: Jakub Tolar; Matthew J O'Shaughnessy; Angela Panoskaltsis-Mortari; Ron T McElmurry; Scott Bell; Megan Riddle; R Scott McIvor; Stephen R Yant; Mark A Kay; Diane Krause; Catherine M Verfaillie; Bruce R Blazar
Host factors that impact the biodistribution and persistence of multipotent adult progenitor cells.
Blood 2006;107(10):4182-8.
2006: Dirk Grimm; Mark A Kay
Therapeutic short hairpin RNA expression in the liver: viral targets and vectors.
Gene therapy 2006;13(6):563-75.
2006: Dirk Grimm; Kusum Pandey; Hiroyuki Nakai; Theresa A Storm; Mark A Kay
Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype.
Journal of virology 2006;80(1):426-39.
2005: Jakub Tolar; Mark J Osborn; Scott Bell; Ron T McElmurry; Lily Xia; Megan Riddle; Angela Panoskaltsis-Mortari; Yuehua Jiang; R Scott McIvor; Christopher H Contag; Stephen R Yant; Mark A Kay; Catherine M Verfaillie; Bruce R Blazar
Real-time in vivo imaging of stem cells following transgenesis by transposition.
Molecular therapy : the journal of the American Society of Gene Therapy 2005;12(1):42-8.
2005: Efren Riu; Dirk Grimm; Zan Huang; Mark A Kay
Increased maintenance and persistence of transgenes by excision of expression cassettes from plasmid sequences in vivo.
Human gene therapy 2005;16(5):558-70.
2005: Anja Ehrhardt; Hui Xu; Zan Huang; Jeffrey A Engler; Mark A Kay
A direct comparison of two nonviral gene therapy vectors for somatic integration: in vivo evaluation of the bacteriophage integrase phiC31 and the Sleeping Beauty transposase.
Molecular therapy : the journal of the American Society of Gene Therapy 2005;11(5):695-706.
2005: Kazuo Ohashi; Hiroyuki Nakai; Linda B Couto; Mark A Kay
Modified infusion procedures affect recombinant adeno-associated virus vector type 2 transduction in the liver.
Human gene therapy 2005;16(3):299-306.
2005: Stephen R Yant; Xiaolin Wu; Yong Huang; Brian Garrison; Shawn M Burgess; Mark A Kay
High-resolution genome-wide mapping of transposon integration in mammals.
Molecular and cellular biology 2005;25(6):2085-94.
2005: Hiroyuki Nakai; Xiaolin Wu; Sally Fuess; Theresa A Storm; David J Munroe; Eugenio Montini; Shawn M Burgess; Markus Grompe; Mark A Kay
Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver.
Journal of virology 2005;79(6):3606-14.
2005: Aaron D Tward; Kirk D Jones; S Yant; Mark A Kay; Rong Wang; John M Bishop
Genomic progression in mouse models for liver tumors.
Cold Spring Harbor symposia on quantitative biology 2005;70():217-24.
2005: Zhi-Ying Chen; Cheng-Yi He; Mark A Kay
Improved production and purification of minicircle DNA vector free of plasmid bacterial sequences and capable of persistent transgene expression in vivo.
Human gene therapy 2005;16(1):126-31.
2005: Dirk Grimm; Kusum Pandey; Mark A Kay
Adeno-associated virus vectors for short hairpin RNA expression.
Methods in enzymology 2005;392():381-405.
2005: Kazuo Ohashi; Jacob M Waugh; Michael D Dake; Takashi Yokoyama; Hiroyuki Kuge; Yoshiyuki Nakajima; Masaki Yamanouchi; Hiroyuki Naka; Akira Yoshioka; Mark A Kay
Liver tissue engineering at extrahepatic sites in mice as a potential new therapy for genetic liver diseases.
Hepatology (Baltimore, Md.) 2005;41(1):132-40.
2005: Hiroyuki Nakai; Sally Fuess; Theresa A Storm; Shin-ichi Muramatsu; Yuko Nara; Mark A Kay
Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice.
Journal of virology 2005;79(1):214-24.
2004: Fumikiyo Ganaha; Kazuo Ohashi; Young Soo Do; Jane Lee; Koji Sugimoto; Hiroki Minamiguchi; Christopher J Elkins; Diba Sameni; Shoreh Modanlou; Mir Ali; Edward Y Kao; Mark A Kay; Jacob M Waugh; Michael D Dake
Efficient inhibition of in-stent restenosis by controlled stent-based inhibition of elastase: a pilot study.
Journal of vascular and interventional radiology : JVIR 2004;15(11):1287-93.
2004: Kazuo Ohashi; Mark A Kay
Extracellular matrix component cotransplantation prolongs survival of heterotopically transplanted human hepatocytes in mice.
Transplantation proceedings 2004;36(8):2469-70.
2004: Stephen R Yant; Julie Park; Yong Huang; Jacob Giehm Mikkelsen; Mark A Kay
Mutational analysis of the N-terminal DNA-binding domain of sleeping beauty transposase: critical residues for DNA binding and hyperactivity in mammalian cells.
Molecular and cellular biology 2004;24(20):9239-47.
2004: D Denison Jenkins; Konrad L Streetz; Monika Tataria; David Sahar; Masashi Kurobe; Michael T Longaker; Mark A Kay; Karl G Sylvester
Donor-derived, liver-specific protein expression after bone marrow transplantation.
Transplantation 2004;78(4):530-6.
2004: Zhi-Ying Chen; Cheng-Yi He; Leonard Meuse; Mark A Kay
Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo.
Gene therapy 2004;11(10):856-64.
2004: Alfred Y Wang; Peter D Peng; Anja Ehrhardt; Theresa A Storm; Mark A Kay
Comparison of adenoviral and adeno-associated viral vectors for pancreatic gene delivery in vivo.
Human gene therapy 2004;15(4):405-13.
2004: Clare E Thomas; Theresa A Storm; Zan Huang; Mark A Kay
Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors.
Journal of virology 2004;78(6):3110-22.
2003: Stephen R Yant; Mark A Kay
Nonhomologous-end-joining factors regulate DNA repair fidelity during Sleeping Beauty element transposition in mammalian cells.
Molecular and cellular biology 2003;23(23):8505-18.
2003: Jacob Giehm Mikkelsen; Stephen R Yant; Leonard Meuse; Zan Huang; Hui Xu; Mark A Kay
Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2003;8(4):654-65.
2003: Anja Ehrhardt; Hui Xu; Aaron Dillow; Dwight A Bellinger; Timothy C Nichols; Mark A Kay
A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia.
Blood 2003;102(7):2403-11.
2003: Dirk Grimm; Shangzhen Zhou; Hiroyuki Nakai; Clare E Thomas; Theresa A Storm; Sally Fuess; Takashi Matsushita; James Allen; Richard Surosky; Michael Lochrie; Leonard Meuse; Alan McClelland; Peter Colosi; Mark A Kay
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy.
Blood 2003;102(7):2412-9.
2003: Yung H Chyung; Peter D Peng; Mark A Kay
System for simultaneous tissue-specific and disease-specific regulation of therapeutic gene expression.
Human gene therapy 2003;14(13):1255-64.
2003: Zhi-Ying Chen; Cheng-Yi He; Anja Ehrhardt; Mark A Kay
Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2003;8(3):495-500.
2003: Bruno B Bordier; Junko Ohkanda; Ping Liu; So-Young Lee; F H Salazar; Patricia L Marion; Kazuo Ohashi; Leonard Meuse; Mark A Kay; John L Casey; Said M Sebti; Andrew D Hamilton; Jeffrey S Glenn
In vivo antiviral efficacy of prenylation inhibitors against hepatitis delta virus.
The Journal of clinical investigation 2003;112(3):407-14.
2003: Anton P McCaffrey; Leonard Meuse; Mobin Karimi; Christopher H Contag; Mark A Kay
A potent and specific morpholino antisense inhibitor of hepatitis C translation in mice.
Hepatology (Baltimore, Md.) 2003;38(2):503-8.
2003: Dirk Grimm; Mark A Kay
From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy.
Current gene therapy 2003;3(4):281-304.
2003: Susana Ortiz-Urda; Qun Lin; Stephen R Yant; Douglas R Keene; Mark A Kay; Paul A Khavari
Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer.
Gene therapy 2003;10(13):1099-104.
2003: Anja Ehrhardt; Hui Xu; Mark A Kay
Episomal persistence of recombinant adenoviral vector genomes during the cell cycle in vivo.
Journal of virology 2003;77(13):7689-95.
2003: Hiroyuki Nakai; Eugenio Montini; Sally Fuess; Theresa A Storm; Markus Grompe; Mark A Kay
AAV serotype 2 vectors preferentially integrate into active genes in mice.
Nature genetics 2003;34(3):297-302.
2003: Hiroyuki Nakai; Theresa A Storm; Sally Fuess; Mark A Kay
Pathways of removal of free DNA vector ends in normal and DNA-PKcs-deficient SCID mouse hepatocytes transduced with rAAV vectors.
Human gene therapy 2003;14(9):871-81.
2003: Dirk Grimm; Mark A Kay; Juergen A Kleinschmidt
Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6.
Molecular therapy : the journal of the American Society of Gene Therapy 2003;7(6):839-50.
2003: Anton P McCaffrey; Hiroyuki Nakai; Kusum Pandey; Zan Huang; Felix H Salazar; Hui Xu; Stefan F Wieland; Patricia L Marion; Mark A Kay
Inhibition of hepatitis B virus in mice by RNA interference.
Nature biotechnology 2003;21(6):639-44.
2003: Clare E Thomas; Anja Ehrhardt; Mark A Kay
Progress and problems with the use of viral vectors for gene therapy.
Nature reviews. Genetics 2003;4(5):346-58.
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: Anton McCaffrey; Mark A Kay; Christopher H Contag
Advancing molecular therapies through in vivo bioluminescent imaging.
Molecular imaging 2003;2(2):75-86.
2003: Anja Ehrhardt; Peter D Peng; Hui Xu; Leonard Meuse; Mark A Kay
Optimization of cis-acting elements for gene expression from nonviral vectors in vivo.
Human gene therapy 2003;14(3):215-25.
2003: Hiroyuki Nakai; Sally Fuess; Theresa A Storm; Leonard Meuse; Mark A Kay
Free DNA ends are essential for concatemerization of synthetic double-stranded adeno-associated virus vector genomes transfected into mouse hepatocytes in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2003;7(1):112-21.
2003: Hiroyuki Nakai; Eugenio Montini; Sally Fuess; Theresa A Storm; Leonard Meuse; Milton Finegold; Markus Grompe; Mark A Kay
Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice.
Molecular therapy : the journal of the American Society of Gene Therapy 2003;7(1):101-11.
2003: Christopher R Sclimenti; Andrew S Neviaser; Edward J Baba; Leonard Meuse; Mark A Kay; Michele P Calos
Epstein-Barr virus vectors provide prolonged robust factor IX expression in mice.
Biotechnology progress 2003;19(1):144-51.
2002: Eugenio Montini; Patrice K Held; Meenakshi Noll; Nicolas Morcinek; Muhsen Al-Dhalimy; Milton Finegold; Stephen R Yant; Mark A Kay; Markus Grompe
In vivo correction of murine tyrosinemia type I by DNA-mediated transposition.
Molecular therapy : the journal of the American Society of Gene Therapy 2002;6(6):759-69.
2002: Anton P McCaffrey; Mark A Kay
A story of mice and men.
Gene therapy 2002;9(23):1563.
2002: Hiroyuki Nakai; Clare E Thomas; Theresa A Storm; Sally Fuess; Sharon Powell; J Fraser Wright; Mark A Kay
A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction.
Journal of virology 2002;76(22):11343-9.
2002: Eric C Olivares; Roger P Hollis; Thomas W Chalberg; Leonard Meuse; Mark A Kay; Michele P Calos
Site-specific genomic integration produces therapeutic Factor IX levels in mice.
Nature biotechnology 2002;20(11):1124-8.
2002: Stephen R Yant; Anja Ehrhardt; Jacob Giehm Mikkelsen; Leonard Meuse; Thao Pham; Mark A Kay
Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo.
Nature biotechnology 2002;20(10):999-1005.
2002: Bruno B Bordier; Patricia L Marion; Kazuo Ohashi; Mark A Kay; Harry B Greenberg; John L Casey; Jeffrey S Glenn
A prenylation inhibitor prevents production of infectious hepatitis delta virus particles.
Journal of virology 2002;76(20):10465-72.
2002: Anton P McCaffrey; Leonard Meuse; Thu-Thao T Pham; Douglas S Conklin; Gregory J Hannon; Mark A Kay
RNA interference in adult mice.
Nature 2002;418(6893):38-9.
2002: Anton P McCaffrey; Kazuo Ohashi; Leonard Meuse; Shiliang Shen; Alissa M Lancaster; Peter J Lukavsky; Peter Sarnow; Mark A Kay
Determinants of hepatitis C translational initiation in vitro, in cultured cells and mice.
Molecular therapy : the journal of the American Society of Gene Therapy 2002;5(6):676-84.
2002: Anja Ehrhardt; Mark A Kay
A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo.
Blood 2002;99(11):3923-30.
2002: Kazuo Ohashi; Frank Park; Mark A Kay
Role of hepatocyte direct hyperplasia in lentivirus-mediated liver transduction in vivo.
Human gene therapy 2002;13(5):653-63.
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: Kazuo Ohashi; Frank Park; Mark A Kay
Hepatocyte transplantation: clinical and experimental application.
Journal of molecular medicine (Berlin, Germany) 2001;79(11):617-30.
2001: Douglas Vollrath; W Feng; Jacque L Duncan; Douglas Yasumura; P M D'Cruz; Aimee V Chappelow; Michael T Matthes; Mark A Kay; Matthew M LaVail
Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk.
Proceedings of the National Academy of Sciences of the United States of America 2001;98(22):12584-9.
2001: Frank Park; Mark A Kay
Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo.
Molecular therapy : the journal of the American Society of Gene Therapy 2001;4(3):164-73.
2001: Stephanie M Stoll; Christopher R Sclimenti; Edward J Baba; Leonard Meuse; Mark A Kay; Michele P Calos
Epstein-Barr virus/human vector provides high-level, long-term expression of alpha1-antitrypsin in mice.
Molecular therapy : the journal of the American Society of Gene Therapy 2001;4(2):122-9.
2001: Hiroyuki Nakai; Stephen R Yant; Theresa A Storm; Sally Fuess; Leonard Meuse; Mark A Kay
Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo.
Journal of virology 2001;75(15):6969-76.
2001: Zhi-Ying Chen; Stephen R Yant; Cheng-Yi He; Leonard Meuse; S Shen; Mark A Kay
Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver.
Molecular therapy : the journal of the American Society of Gene Therapy 2001;3(3):403-10.
2001: Kazuo Ohashi; Leonard Meuse; R Schwall; Mark A Kay
cMet activation allows persistent engraftment of ectopically transplanted xenogenic human hepatocytes in mice.
Transplantation proceedings 2001;33(1-2):587-8.
2001: Mark A Kay; J C Glorioso; L Naldini
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics.
Nature medicine 2001;7(1):33-40.
2000: Hiroyuki Nakai; Theresa A Storm; Mark A Kay
Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo.
Journal of virology 2000;74(20):9451-63.
2000: Frank Park; Kazuo Ohashi; Mark A Kay
Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver.
Blood 2000;96(3):1173-6.
2000: Carol H Miao; K Ohashi; GA Patijn; Leonard Meuse; Xin Ye; Arthur R Thompson; Mark A Kay
Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro.
Molecular therapy : the journal of the American Society of Gene Therapy 2000;1(6):522-32.
2000: Stephen R Yant; Leonard Meuse; W Chiu; Z Ivics; Z Izsvak; Mark A Kay
Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system.
Nature genetics 2000;25(1):35-41.
2000: Hiroyuki Nakai; Theresa A Storm; Mark A Kay
Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors.
Nature biotechnology 2000;18(5):527-32.
2000: Carol H Miao; Hiroyuki Nakai; Arthur R Thompson; T A Storm; W Chiu; Richard O Snyder; Mark A Kay
Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction.
Journal of virology 2000;74(8):3793-803.
2000: Kazuo Ohashi; Patricia L Marion; Hiroyuki Nakai; Leonard Meuse; John M Cullen; Bruno B Bordier; R Schwall; Harry B Greenberg; Jeffrey S Glenn; Mark A Kay
Sustained survival of human hepatocytes in mice: A model for in vivo infection with human hepatitis B and hepatitis delta viruses.
Nature medicine 2000;6(3):327-31.
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.
2000: André Lieber; Mark A Kay; Zong-Yi Li
Nuclear import of moloney murine leukemia virus DNA mediated by adenovirus preterminal protein is not sufficient for efficient retroviral transduction in nondividing cells.
Journal of virology 2000;74(2):721-34.
2000: Frank Park; Kazuo Ohashi; W Chiu; L Naldini; Mark A Kay
Efficient lentiviral transduction of liver requires cell cycling in vivo.
Nature genetics 2000;24(1):49-52.
1999: André Lieber; Dirk S Steinwaerder; Cheryl A Carlson; Mark A Kay
Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes.
Journal of virology 1999;73(11):9314-24.
1999: K Zen; Aly Karsan; April Stempien-Otero; E Yee; Joan C Tupper; Xianwu Li; T Eunson; Mark A Kay; Christopher B Wilson; Robert K Winn; John M Harlan
NF-kappaB activation is required for human endothelial survival during exposure to tumor necrosis factor-alpha but not to interleukin-1beta or lipopolysaccharide.
The Journal of biological chemistry 1999;274(40):28808-15.
1999: David W Russell; Mark A Kay
Adeno-associated virus vectors and hematology.
Blood 1999;94(3):864-74.
1999: Hiroyuki Nakai; Y Iwaki; Mark A Kay; Linda B Couto
Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver.
Journal of virology 1999;73(7):5438-47.
1999: D B Schowalter; C L Himeda; BL Winther; C B Wilson; Mark A Kay
Implication of interfering antibody formation and apoptosis as two different mechanisms leading to variable duration of adenovirus-mediated transgene expression in immune-competent mice.
Journal of virology 1999;73(6):4755-66.
1999: GA Patijn; Mark A Kay
Hepatic gene therapy using adeno-associated virus vectors.
Seminars in liver disease 1999;19(1):61-9.
1999: Richard O Snyder; C Miao; Leonard Meuse; J Tubb; Brian A Donahue; Hui-Feng Lin; Darrel W Stafford; S Patel; Arthur R Thompson; Timothy C Nichols; Marjorie S Read; Dwight A Bellinger; Kenneth Brinkhous; Mark A Kay
Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors.
Nature medicine 1999;5(1):64-70.
1998: H Mizuguchi; Mark A Kay
Efficient construction of a recombinant adenovirus vector by an improved in vitro ligation method.
Human gene therapy 1998;9(17):2577-83.
1998: André Lieber; Cheng-Yi He; Leonard Meuse; Charis L Himeda; C Wilson; Mark A Kay
Inhibition of NF-kappaB activation in combination with bcl-2 expression allows for persistence of first-generation adenovirus vectors in the mouse liver.
Journal of virology 1998;72(11):9267-77.
1998: GA Patijn; André Lieber; DB Schowalter; R Schwall; Mark A Kay
Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice.
Hepatology (Baltimore, Md.) 1998;28(3):707-16.
1998: GA Patijn; O T Terpstra; Mark A Kay
Method for continuous infusion into the portal vein of mice.
Laboratory animal science 1998;48(4):379-83.
1998: Mark A Kay
Hepatic gene therapy for haemophilia B.
Haemophilia : the official journal of the World Federation of Hemophilia 1998;4(4):389-92.
1998: GA Patijn; André Lieber; Leonard Meuse; BL Winther; Mark A Kay
High-efficiency retrovirus-mediated gene transfer into the livers of mice.
Human gene therapy 1998;9(10):1449-56.
1998: Carol H Miao; Richard O Snyder; DB Schowalter; GA Patijn; Brian A Donahue; BL Winther; Mark A Kay
The kinetics of rAAV integration in the liver.
Nature genetics 1998;19(1):13-5.
1997: A Lieber; C Y He; Mark A Kay
Adenoviral preterminal protein stabilizes mini-adenoviral genomes in vitro and in vivo.
Nature biotechnology 1997;15(13):1383-7.
1997: Mark A Kay; D Liu; P M Hoogerbrugge
Gene therapy.
Proceedings of the National Academy of Sciences of the United States of America 1997;94(24):12744-6.
1997: James E Nelson; Mark A Kay
Persistence of recombinant adenovirus in vivo is not dependent on vector DNA replication.
Journal of virology 1997;71(11):8902-7.
1997: André Lieber; Cheng-Yi He; Leonard Meuse; DB Schowalter; Irina Kirillova; BL Winther; Mark A Kay
The role of Kupffer cell activation and viral gene expression in early liver toxicity after infusion of recombinant adenovirus vectors.
Journal of virology 1997;71(11):8798-807.
1997: DB Schowalter; Mark A Kay
Gene therapy: a status report.
Pediatric annals 1997;26(9):562-8.
1997: DB Schowalter; Leonard Meuse; Christopher B Wilson; Peter Linsley; Mark A Kay
Constitutive expression of murine CTLA4Ig from a recombinant adenovirus vector results in prolonged transgene expression.
Gene therapy 1997;4(8):853-60.
1997: Richard O Snyder; Carol H Miao; GA Patijn; SK Spratt; O Danos; Dea Nagy; A M Gown; BL Winther; Leonard Meuse; LK Cohen; Arthur R Thompson; Mark A Kay
Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors.
Nature genetics 1997;16(3):270-6.
1997: Mark A Kay
Adenoviral vectors for hepatic gene transfer in animals.
Chest 1997;111(6 Suppl):138S-142S.
1997: Mark A Kay; Leonard Meuse; A M Gown; Peter Linsley; Diane L Hollenbaugh; Alejandro Aruffo; Hans D Ochs; Christopher B Wilson
Transient immunomodulation with anti-CD40 ligand antibody and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver.
Proceedings of the National Academy of Sciences of the United States of America 1997;94(9):4686-91.
1997: DB Schowalter; Julie Tubb; M Liu; Christopher B Wilson; Mark A Kay
Heterologous expression of adenovirus E3-gp19K in an E1a-deleted adenovirus vector inhibits MHC I expression in vitro, but does not prolong transgene expression in vivo.
Gene therapy 1997;4(4):351-60.
1997: M J Vrancken Peeters; GA Patijn; André Lieber; J Perkins; Mark A Kay
Expansion of donor hepatocytes after recombinant adenovirus-induced liver regeneration in mice.
Hepatology (Baltimore, Md.) 1997;25(4):884-8.
1997: Mark A Kay; Nelson Fausto
Liver regeneration: prospects for therapy based on new technologies.
Molecular medicine today 1997;3(3):108-15.
1996: André Lieber; Cheng-Yi He; Irina Kirillova; Mark A Kay
Recombinant adenoviruses with large deletions generated by Cre-mediated excision exhibit different biological properties compared with first-generation vectors in vitro and in vivo.
Journal of virology 1996;70(12):8944-60.
1996: André Lieber; Cheng-Yi He; Stephen Polyak; David R Gretch; Dana Barr; Mark A Kay
Elimination of hepatitis C virus RNA in infected human hepatocytes by adenovirus-mediated expression of ribozymes.
Journal of virology 1996;70(12):8782-91.
1996: Arthur R Thompson; Mark A Kay
Nonviral gene transfer to the liver.
Hepatology (Baltimore, Md.) 1996;24(6):1541-2.
1996: V Sandig; Peter Löser; A Lieber; Mark A Kay; M Strauss
HBV-derived promoters direct liver-specific expression of an adenovirally transduced LDL receptor gene.
Gene therapy 1996;3(11):1002-9.
1996: M J Peeters; GA Patijn; André Lieber; Leonard Meuse; Mark A Kay
Adenovirus-mediated hepatic gene transfer in mice: comparison of intravascular and biliary administration.
Human gene therapy 1996;7(14):1693-9.
1996: André Lieber; Mark A Kay
Adenovirus-mediated expression of ribozymes in mice.
Journal of virology 1996;70(5):3153-8.
1996: M L Liu; BL Winther; Mark A Kay
Pseudotransduction of hepatocytes by using concentrated pseudotyped vesicular stomatitis virus G glycoprotein (VSV-G)-Moloney murine leukemia virus-derived retrovirus vectors: comparison of VSV-G and amphotropic vectors for hepatic gene transfer.
Journal of virology 1996;70(4):2497-502.
1996: M J Vrancken Peeters; A L Perkins; Mark A Kay
Method for multiple portal vein infusions in mice: quantitation of adenovirus-mediated hepatic gene transfer.
BioTechniques 1996;20(2):278-85.
1995: Mark A Kay; Ai-Xuan Holterman; Leonard Meuse; Allen M Gown; Hans D Ochs; Peter Linsley; Christopher B Wilson
Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration.
Nature genetics 1995;11(2):191-7.
1995: C Wilson; Mark A Kay
Immunomodulation to enhance gene therapy.
Nature medicine 1995;1(9):887-9.
1995: Bingliang Fang; Randy C Eisensmith; H Wang; Mark A Kay; R E Cross; C N Landen; G Gordon; Dwight A Bellinger; Marjorie S Read; P C Hu
Gene therapy for hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression.
Human gene therapy 1995;6(8):1039-44.
1995: André Lieber; M J Peeters; A Gown; J Perkins; Mark A Kay
A modified urokinase plasminogen activator induces liver regeneration without bleeding.
Human gene therapy 1995;6(8):1029-37.
1995: André Lieber; M J Vrancken Peeters; Leonard Meuse; Nelson Fausto; J Perkins; Mark A Kay
Adenovirus-mediated urokinase gene transfer induces liver regeneration and allows for efficient retrovirus transduction of hepatocytes in vivo.
Proceedings of the National Academy of Sciences of the United States of America 1995;92(13):6210-4.
1995: A Scaria; D T Curiel; Mark A Kay
Complementation of a human adenovirus early region 4 deletion mutant in 293 cells using adenovirus-polylysine-DNA complexes.
Gene therapy 1995;2(4):295-8.
1995: Mark A Kay; F Graham; F Leland; SL Woo
Therapeutic serum concentrations of human alpha-1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes.
Hepatology (Baltimore, Md.) 1995;21(3):815-9.
1995: Dana Barr; J Tubb; D Ferguson; A Scaria; André Lieber; C Wilson; J Perkins; Mark A Kay
Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains.
Gene therapy 1995;2(2):151-5.
1995: Mark A Kay
Hepatic gene therapy for hemophilia B.
Advances in experimental medicine and biology 1995;386():229-34.
1995: André Lieber; M J Vrancken Peeters; Mark A Kay
Adenovirus-mediated transfer of the amphotropic retrovirus receptor cDNA increases retroviral transduction in cultured cells.
Human gene therapy 1995;6(1):5-11.
1994: Mark A Kay; C N Landen; S R Rothenberg; L A Taylor; F Leland; S Wiehle; Bingliang Fang; Dwight A Bellinger; Milton Finegold; Arthur R Thompson
In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs.
Proceedings of the National Academy of Sciences of the United States of America 1994;91(6):2353-7.
1993: R J Cristiano; Louis C Smith; Mark A Kay; Bill R Brinkley; SL Woo
Hepatic gene therapy: efficient gene delivery and expression in primary hepatocytes utilizing a conjugated adenovirus-DNA complex.
Proceedings of the National Academy of Sciences of the United States of America 1993;90(24):11548-52.
1993: Mark A Kay; S Rothenberg; C N Landen; Dwight A Bellinger; F Leland; C Toman; Milton Finegold; Arthur R Thompson; Marjorie S Read; Kenneth Brinkhous
In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs.
Science (New York, N.Y.) 1993;262(5130):117-9.
1993: T M Kolodka; Milton Finegold; Mark A Kay; SL Woo
Hepatic gene therapy: efficient retroviral-mediated gene transfer into rat hepatocytes in vivo.
Somatic cell and molecular genetics 1993;19(5):491-7.
1993: Q Li; Mark A Kay; Milton Finegold; L D Stratford-Perricaudet; SL Woo
Assessment of recombinant adenoviral vectors for hepatic gene therapy.
Human gene therapy 1993;4(4):403-9.
1992: Mark A Kay; Q Li; T J Liu; F Leland; C Toman; Milton Finegold; SL Woo
Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo.
Human gene therapy 1992;3(6):641-7.
1992: Mark A Kay; P Baley; S Rothenberg; F Leland; L Fleming; K P Ponder; T Liu; Milton Finegold; G Darlington; W Pokorny
Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes.
Proceedings of the National Academy of Sciences of the United States of America 1992;89(1):89-93.
1992: Mark A Kay; K P Ponder; SL Woo
Human gene therapy: present and future.
Breast cancer research and treatment 1992;21(2):83-93.
1992: T J Liu; Mark A Kay; G J Darlington; SL Woo
Reconstitution of enzymatic activity in hepatocytes of phenylalanine hydroxylase-deficient mice.
Somatic cell and molecular genetics 1992;18(1):89-96.