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Volumn 10, Issue 5, 2008, Pages 464-470

Factoring nonviral gene therapy into a cure for hemophilia A

Author keywords

Factor VIII; Gene therapy; Hemophilia A; Inhibitor antibodies; Nonviral DNA delivery; Plasmid DNA

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 9; INTEGRASE; LIPOSOME; NAKED DNA; NANOPARTICLE; NEUTRALIZING ANTIBODY; PARVOVIRUS VECTOR; PLASMID DNA; POLYETHYLENEIMINE; POLYMER; RECOMBINANT BLOOD CLOTTING FACTOR 7A; RECOMBINANT BLOOD CLOTTING FACTOR 8; TRANSPOSASE; VIRUS VECTOR;

EID: 53249093884     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review
Times cited : (4)

References (64)
  • 2
    • 33846453333 scopus 로고    scopus 로고
    • Shi Q, Wilcox DA, Fahs SA, Fang J, Johnson BD, Du LM, Desai D, Montgomery RR: Lentivirus-mediated platelet-derived Factor VIII gene therapy in murine haemophilia A. J Thromb Haemost (2007) 5(2):352-361. •• This represents an elegant study demonstrating phenotypic cure in hemophilia A mice, despite high-titer antibodies against human Factor VIII. Factor VIII production was limited to platelets and released at the site of injury.
    • Shi Q, Wilcox DA, Fahs SA, Fang J, Johnson BD, Du LM, Desai D, Montgomery RR: Lentivirus-mediated platelet-derived Factor VIII gene therapy in murine haemophilia A. J Thromb Haemost (2007) 5(2):352-361. •• This represents an elegant study demonstrating phenotypic cure in hemophilia A mice, despite high-titer antibodies against human Factor VIII. Factor VIII production was limited to platelets and released at the site of injury.
  • 3
    • 28244484911 scopus 로고    scopus 로고
    • The promise and challenges of bioengineered recombinant clotting factors
    • Pipe SW: The promise and challenges of bioengineered recombinant clotting factors. J Thromb Haemost (2005) 3(8):1692-1701.
    • (2005) J Thromb Haemost , vol.3 , Issue.8 , pp. 1692-1701
    • Pipe, S.W.1
  • 4
    • 24944585648 scopus 로고    scopus 로고
    • Gene therapy for hemophilia: An imperative to succeed
    • Hough C, Lillicrap D: Gene therapy for hemophilia: An imperative to succeed. J Thromb Haemost (2005) 3(6):1195-1205.
    • (2005) J Thromb Haemost , vol.3 , Issue.6 , pp. 1195-1205
    • Hough, C.1    Lillicrap, D.2
  • 5
    • 0035822005 scopus 로고    scopus 로고
    • Nonviral transfer of the gene encoding coagulation Factor VIII in patients with severe hemophilia A
    • This ex vivo strategy is currently the only nonviral gene therapy study in hemophilia A patients. The clinical trial was initially successful, but did not result in long-term expression of FVIII, ••
    • Roth DA, Tawa NE Jr, O'Brien JM, Treco DA, Selden RF: Nonviral transfer of the gene encoding coagulation Factor VIII in patients with severe hemophilia A. N Engl J Med (2001) 344(23):1735-1742. •• This ex vivo strategy is currently the only nonviral gene therapy study in hemophilia A patients. The clinical trial was initially successful, but did not result in long-term expression of FVIII.
    • (2001) N Engl J Med , vol.344 , Issue.23 , pp. 1735-1742
    • Roth, D.A.1    Tawa Jr, N.E.2    O'Brien, J.M.3    Treco, D.A.4    Selden, R.F.5
  • 6
    • 0141541822 scopus 로고    scopus 로고
    • Infectious risks of blood transfusion
    • Uhl L: Infectious risks of blood transfusion. Curr Hematol Rep (2002) 1(2):156-162.
    • (2002) Curr Hematol Rep , vol.1 , Issue.2 , pp. 156-162
    • Uhl, L.1
  • 8
    • 33745621052 scopus 로고    scopus 로고
    • Immunomodulation of transgene responses following naked DNA transfer of human Factor VIII into hemophilia A mice
    • Miao CH, Ye P, Thompson AR, Rawlings DJ, Ochs HD: Immunomodulation of transgene responses following naked DNA transfer of human Factor VIII into hemophilia A mice. Blood (2006) 108(1):19-27.
    • (2006) Blood , vol.108 , Issue.1 , pp. 19-27
    • Miao, C.H.1    Ye, P.2    Thompson, A.R.3    Rawlings, D.J.4    Ochs, H.D.5
  • 9
    • 0038209049 scopus 로고    scopus 로고
    • Recombinant activated Factor VII: A new weapon in the fight against hemorrhage
    • Branch DW, Rodgers GM: Recombinant activated Factor VII: A new weapon in the fight against hemorrhage. Obstet Gynecol (2003) 101(6):1155-1156.
    • (2003) Obstet Gynecol , vol.101 , Issue.6 , pp. 1155-1156
    • Branch, D.W.1    Rodgers, G.M.2
  • 10
    • 34247855110 scopus 로고    scopus 로고
    • Nonviral gene delivery: What we know and what is next
    • Gao X, Kim KS, Liu D: Nonviral gene delivery: What we know and what is next. AAPSJ (2007) 9(1):E92-E104.
    • (2007) AAPSJ , vol.9 , Issue.1
    • Gao, X.1    Kim, K.S.2    Liu, D.3
  • 11
    • 34247862426 scopus 로고    scopus 로고
    • Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment
    • Pierce GF, Lillicrap D, Pipe SW, Vandendriessche T: Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment. J Thromb Haemost (2007) 5(5):901-906.
    • (2007) J Thromb Haemost , vol.5 , Issue.5 , pp. 901-906
    • Pierce, G.F.1    Lillicrap, D.2    Pipe, S.W.3    Vandendriessche, T.4
  • 15
    • 0037790710 scopus 로고    scopus 로고
    • Molecular basis of the inflammatory response to adenovirus vectors
    • Liu Q, Muruve DA: Molecular basis of the inflammatory response to adenovirus vectors. Gene Ther (2003) 10(11):935-940.
    • (2003) Gene Ther , vol.10 , Issue.11 , pp. 935-940
    • Liu, Q.1    Muruve, D.A.2
  • 16
    • 0038069517 scopus 로고    scopus 로고
    • The role of capsid-endothelial interactions in the innate immune response to adenovirus vectors
    • Liu Q, Zaiss AK, Colarusso P, Patel K, Haljan G, Wickham TJ, Muruve DA: The role of capsid-endothelial interactions in the innate immune response to adenovirus vectors. Hum Gene Ther (2003) 14(7):627-643.
    • (2003) Hum Gene Ther , vol.14 , Issue.7 , pp. 627-643
    • Liu, Q.1    Zaiss, A.K.2    Colarusso, P.3    Patel, K.4    Haljan, G.5    Wickham, T.J.6    Muruve, D.A.7
  • 19
    • 30344479961 scopus 로고    scopus 로고
    • Determination of specific CD4 and CD8 T cell epitopes after AAV2- and AAV8-hF.IX gene therapy
    • Chen J, Wu Q, Yang P, Hsu HC, Mountz JD: Determination of specific CD4 and CD8 T cell epitopes after AAV2- and AAV8-hF.IX gene therapy. Mol Ther (2006) 13(2):260-269.
    • (2006) Mol Ther , vol.13 , Issue.2 , pp. 260-269
    • Chen, J.1    Wu, Q.2    Yang, P.3    Hsu, H.C.4    Mountz, J.D.5
  • 20
    • 33344466244 scopus 로고    scopus 로고
    • Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice
    • Scallan CD, Jiang H, Liu T, Patarroyo-White S, Sommer JM, Zhou S, Couto LB, Pierce GF: Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice. Blood (2006) 107(5):1810-1817.
    • (2006) Blood , vol.107 , Issue.5 , pp. 1810-1817
    • Scallan, C.D.1    Jiang, H.2    Liu, T.3    Patarroyo-White, S.4    Sommer, J.M.5    Zhou, S.6    Couto, L.B.7    Pierce, G.F.8
  • 22
    • 33746733616 scopus 로고    scopus 로고
    • Gene therapy for hemophilia
    • Ponder KP: Gene therapy for hemophilia. Curr Opin Hematol (2006) 13(5):301-307.
    • (2006) Curr Opin Hematol , vol.13 , Issue.5 , pp. 301-307
    • Ponder, K.P.1
  • 23
    • 33749337200 scopus 로고    scopus 로고
    • Genetic engineering for haemophilia A
    • Gan SU, Kon OL, Calne RY: Genetic engineering for haemophilia A. Expert Opin Biol Ther (2006) 6(10):1023-1030.
    • (2006) Expert Opin Biol Ther , vol.6 , Issue.10 , pp. 1023-1030
    • Gan, S.U.1    Kon, O.L.2    Calne, R.Y.3
  • 24
    • 33746140392 scopus 로고    scopus 로고
    • Review of Epstein-Barr virus and post-transplant lymphoproliferative disorder post-solid organ transplantation
    • Lim WH, Russ GR, Coates PT: Review of Epstein-Barr virus and post-transplant lymphoproliferative disorder post-solid organ transplantation. Nephrology (Carlton) (2006) 11(4):355-366.
    • (2006) Nephrology (Carlton) , vol.11 , Issue.4 , pp. 355-366
    • Lim, W.H.1    Russ, G.R.2    Coates, P.T.3
  • 25
    • 0038054338 scopus 로고    scopus 로고
    • AAV serotype 2 vectors preferentially integrate into active genes in mice
    • Nakai H, Montini E, Fuess S, Storm TA, Grompe M, Kay MA: AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet (2003) 34(3):297-302.
    • (2003) Nat Genet , vol.34 , Issue.3 , pp. 297-302
    • Nakai, H.1    Montini, E.2    Fuess, S.3    Storm, T.A.4    Grompe, M.5    Kay, M.A.6
  • 26
    • 0041315884 scopus 로고    scopus 로고
    • Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo
    • Chen ZY, He CY, Ehrhardt A, Kay MA: Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther (2003) 8(3):495-500.
    • (2003) Mol Ther , vol.8 , Issue.3 , pp. 495-500
    • Chen, Z.Y.1    He, C.Y.2    Ehrhardt, A.3    Kay, M.A.4
  • 27
    • 0031688612 scopus 로고    scopus 로고
    • Inflammatory responses following direct injection of plasmid DNA into skeletal muscle
    • McMahon JM, Wells KE, Bamfo JE, Cartwright MA, Wells DJ: Inflammatory responses following direct injection of plasmid DNA into skeletal muscle. Gene Ther (1998) 5(9):1283-1290.
    • (1998) Gene Ther , vol.5 , Issue.9 , pp. 1283-1290
    • McMahon, J.M.1    Wells, K.E.2    Bamfo, J.E.3    Cartwright, M.A.4    Wells, D.J.5
  • 28
    • 33746238237 scopus 로고    scopus 로고
    • Mechanism of plasmid delivery by hydrodynamic tail vein injection. II. Morphological studies
    • Budker VG, Subbotin VM, Budker T, Sebestyén MG, Zhang G, Wolff JA: Mechanism of plasmid delivery by hydrodynamic tail vein injection. II. Morphological studies. J Gene Med (2006) 8(7):874-888.
    • (2006) J Gene Med , vol.8 , Issue.7 , pp. 874-888
    • Budker, V.G.1    Subbotin, V.M.2    Budker, T.3    Sebestyén, M.G.4    Zhang, G.5    Wolff, J.A.6
  • 29
    • 33845973756 scopus 로고    scopus 로고
    • Structural impact of hydrodynamic injection on mouse liver
    • Suda T, Gao X, Stolz DB, Liu D: Structural impact of hydrodynamic injection on mouse liver. Gene Ther (2007) 14(2):129-137.
    • (2007) Gene Ther , vol.14 , Issue.2 , pp. 129-137
    • Suda, T.1    Gao, X.2    Stolz, D.B.3    Liu, D.4
  • 31
    • 33846022383 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: Hydrodynamic gene delivery
    • An important and comprehensive review of the hydrodynamic method for nonviral gene delivery, ••
    • Herweijer H, Wolff JA: Gene therapy progress and prospects: Hydrodynamic gene delivery. Gene Ther (2007) 14(2):99-107. •• An important and comprehensive review of the hydrodynamic method for nonviral gene delivery.
    • (2007) Gene Ther , vol.14 , Issue.2 , pp. 99-107
    • Herweijer, H.1    Wolff, J.A.2
  • 33
    • 33846922049 scopus 로고    scopus 로고
    • Pig liver gene therapy by noninvasive interventionist catheterism
    • This successful large-animal study demonstrated a modification of hydrodynamic injection in pigs for delivery of nonviral DNA to the liver. The study suggested that the method could be further manipulated for use in humans, •
    • Aliño SF, Herrero MJ, Noguera I, Dasí F, Sánchez M: Pig liver gene therapy by noninvasive interventionist catheterism. Gene Ther (2007) 14(4):334-343. • This successful large-animal study demonstrated a modification of hydrodynamic injection in pigs for delivery of nonviral DNA to the liver. The study suggested that the method could be further manipulated for use in humans.
    • (2007) Gene Ther , vol.14 , Issue.4 , pp. 334-343
    • Aliño, S.F.1    Herrero, M.J.2    Noguera, I.3    Dasí, F.4    Sánchez, M.5
  • 34
    • 0031753867 scopus 로고    scopus 로고
    • Gene transfer into muscle by electroporation in vivo
    • Aihara H, Miyazaki J: Gene transfer into muscle by electroporation in vivo. Nat Biotechnol (1998) 16(9):867-870.
    • (1998) Nat Biotechnol , vol.16 , Issue.9 , pp. 867-870
    • Aihara, H.1    Miyazaki, J.2
  • 35
    • 0041375537 scopus 로고    scopus 로고
    • Electroporation as a method for high-level nonviral gene transfer to the lung
    • Dean DA, Machado-Aranda D, Blair-Parks K, Yeldandi AV, Young JL: Electroporation as a method for high-level nonviral gene transfer to the lung. Gene Ther (2003) 10(18):1608-1615.
    • (2003) Gene Ther , vol.10 , Issue.18 , pp. 1608-1615
    • Dean, D.A.1    Machado-Aranda, D.2    Blair-Parks, K.3    Yeldandi, A.V.4    Young, J.L.5
  • 36
    • 3042651120 scopus 로고    scopus 로고
    • Electroporation for gene transfer to skeletal muscles: Current status
    • McMahon JM, Wells DJ: Electroporation for gene transfer to skeletal muscles: Current status. BioDrugs (2004) 18(3):155-165.
    • (2004) BioDrugs , vol.18 , Issue.3 , pp. 155-165
    • McMahon, J.M.1    Wells, D.J.2
  • 37
    • 0034691505 scopus 로고    scopus 로고
    • Ultrasound facilitates transduction of naked plasmid DNA into colon carcinoma cells in vitro and in vivo
    • Manome Y, Nakamura M, Ohno T, Furuhata H: Ultrasound facilitates transduction of naked plasmid DNA into colon carcinoma cells in vitro and in vivo. Hum Gene Ther (2000) 11(11):1521-1528.
    • (2000) Hum Gene Ther , vol.11 , Issue.11 , pp. 1521-1528
    • Manome, Y.1    Nakamura, M.2    Ohno, T.3    Furuhata, H.4
  • 38
    • 0036227368 scopus 로고    scopus 로고
    • Development of safe and efficient novel nonviral gene transfer using ultrasound: Enhancement of transfection efficiency of naked plasmid DNA in skeletal muscle
    • Taniyama Y, Tachibana K, Hiraoka K, Aoki M, Yamamoto S, Matsumoto K, Nakamura T, Ogihara T, Kaneda Y, Morishita R: Development of safe and efficient novel nonviral gene transfer using ultrasound: Enhancement of transfection efficiency of naked plasmid DNA in skeletal muscle. Gene Ther (2002) 9(6):372-380.
    • (2002) Gene Ther , vol.9 , Issue.6 , pp. 372-380
    • Taniyama, Y.1    Tachibana, K.2    Hiraoka, K.3    Aoki, M.4    Yamamoto, S.5    Matsumoto, K.6    Nakamura, T.7    Ogihara, T.8    Kaneda, Y.9    Morishita, R.10
  • 39
    • 0033017213 scopus 로고    scopus 로고
    • DNA delivery from polymer matrices for tissue engineering
    • Shea LD, Smiley E, Bonadio J, Mooney DJ: DNA delivery from polymer matrices for tissue engineering. Nat Biotechnol (1999) 17(6):551-554.
    • (1999) Nat Biotechnol , vol.17 , Issue.6 , pp. 551-554
    • Shea, L.D.1    Smiley, E.2    Bonadio, J.3    Mooney, D.J.4
  • 40
    • 0242352521 scopus 로고    scopus 로고
    • Substrate-mediated DNA delivery: Role of the cationic polymer structure and extent of modification
    • Segura T, Volk MJ, Shea LD: Substrate-mediated DNA delivery: Role of the cationic polymer structure and extent of modification. J Control Release (2003) 93(1):69-84.
    • (2003) J Control Release , vol.93 , Issue.1 , pp. 69-84
    • Segura, T.1    Volk, M.J.2    Shea, L.D.3
  • 41
    • 0037426720 scopus 로고    scopus 로고
    • Controllable delivery of non-viral DNA from porous scaffolds
    • Jang JH, Shea LD: Controllable delivery of non-viral DNA from porous scaffolds. J Control Release (2003) 86(1):157-168.
    • (2003) J Control Release , vol.86 , Issue.1 , pp. 157-168
    • Jang, J.H.1    Shea, L.D.2
  • 42
    • 0036000157 scopus 로고    scopus 로고
    • Surface-tethered DNA complexes for enhanced gene delivery
    • Segura T, Shea LD: Surface-tethered DNA complexes for enhanced gene delivery. Bioconjug Chem (2002) 13(3):621-629.
    • (2002) Bioconjug Chem , vol.13 , Issue.3 , pp. 621-629
    • Segura, T.1    Shea, L.D.2
  • 43
    • 3042814407 scopus 로고    scopus 로고
    • Controlled release systems for DNA delivery
    • Pannier AK, Shea LD: Controlled release systems for DNA delivery. Mol Ther (2004) 10(1):19-26.
    • (2004) Mol Ther , vol.10 , Issue.1 , pp. 19-26
    • Pannier, A.K.1    Shea, L.D.2
  • 44
    • 2342597890 scopus 로고    scopus 로고
    • Nonviral gene therapy approaches to hemophilia
    • Gomez-Vargas A, Hortelano G: Nonviral gene therapy approaches to hemophilia. Semin Thromb Hemost (2004) 30(2):197-204.
    • (2004) Semin Thromb Hemost , vol.30 , Issue.2 , pp. 197-204
    • Gomez-Vargas, A.1    Hortelano, G.2
  • 45
    • 0036842502 scopus 로고    scopus 로고
    • Olivares EC, Hollis RP, Chalberg TW, Meuse L, Kay MA, Calos MP: Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol (2002) 20(11):1124-1128. • In this study, Factor IX expression was achieved in mice using the φC31 integrase system and demonstrated the specificity of this integrase for pseudo attP sites in the murine genome.
    • Olivares EC, Hollis RP, Chalberg TW, Meuse L, Kay MA, Calos MP: Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol (2002) 20(11):1124-1128. • In this study, Factor IX expression was achieved in mice using the φC31 integrase system and demonstrated the specificity of this integrase for pseudo attP sites in the murine genome.
  • 46
    • 0141539412 scopus 로고    scopus 로고
    • High-level Factor VIII gene expression in vivo achieved by nonviral liver-specific gene therapy vectors
    • Miao CH, Ye X, Thompson AR: High-level Factor VIII gene expression in vivo achieved by nonviral liver-specific gene therapy vectors. Hum Gene Ther (2003) 14(14):1297-1305.
    • (2003) Hum Gene Ther , vol.14 , Issue.14 , pp. 1297-1305
    • Miao, C.H.1    Ye, X.2    Thompson, A.R.3
  • 47
    • 0034103009 scopus 로고    scopus 로고
    • Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system
    • Yant SR, Meuse L, Chiu W, Ivics Z, Izsvak Z, Kay MA: Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system. Nat Genet (2000) 25(1):35-41.
    • (2000) Nat Genet , vol.25 , Issue.1 , pp. 35-41
    • Yant, S.R.1    Meuse, L.2    Chiu, W.3    Ivics, Z.4    Izsvak, Z.5    Kay, M.A.6
  • 48
    • 33645882508 scopus 로고    scopus 로고
    • Sustained FVIII expression and phenotypic correction of hemophilia A in neonatal mice using an endothelial-targeted sleeping beauty transposon
    • Liu L, Mah C, Fletcher BS: Sustained FVIII expression and phenotypic correction of hemophilia A in neonatal mice using an endothelial-targeted sleeping beauty transposon. Mol Ther (2006) 13(5):1006-1015.
    • (2006) Mol Ther , vol.13 , Issue.5 , pp. 1006-1015
    • Liu, L.1    Mah, C.2    Fletcher, B.S.3
  • 50
    • 0036760213 scopus 로고    scopus 로고
    • Defining critical windows in the development of the human immune system
    • West LJ: Defining critical windows in the development of the human immune system. Hum Exp Toxicol (2002) 21(9-10):499-505.
    • (2002) Hum Exp Toxicol , vol.21 , Issue.9-10 , pp. 499-505
    • West, L.J.1
  • 51
    • 0036582006 scopus 로고    scopus 로고
    • Developmental aspects of immunomodulation: Exploiting the immature immune system for organ transplantation
    • West LJ: Developmental aspects of immunomodulation: Exploiting the immature immune system for organ transplantation. Transpl Immunol (2002) 9(2-4):149-153.
    • (2002) Transpl Immunol , vol.9 , Issue.2-4 , pp. 149-153
    • West, L.J.1
  • 52
    • 20144386644 scopus 로고    scopus 로고
    • Ohlfest JR, Frandsen JL, Fritz S, Lobitz PD, Perkinson SG, Clark KJ, Nelsestuen G, Key NS, McIvor RS, Hackett PB, Largaespada DA: Phenotypic correction and long-term expression of Factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system. Blood (2005) 105(7):2691-2698. • This study demonstrated that immunotolerization of neonatal hemophilia A mice decreased the subsequent immune response to Factor VIII gene therapy.
    • Ohlfest JR, Frandsen JL, Fritz S, Lobitz PD, Perkinson SG, Clark KJ, Nelsestuen G, Key NS, McIvor RS, Hackett PB, Largaespada DA: Phenotypic correction and long-term expression of Factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system. Blood (2005) 105(7):2691-2698. • This study demonstrated that immunotolerization of neonatal hemophilia A mice decreased the subsequent immune response to Factor VIII gene therapy.
  • 53
    • 22144464502 scopus 로고    scopus 로고
    • φC31 integrase confers genomic integration and long-term transgene expression in rat retina
    • Chalberg TW, Genise HL, Vollrath D, Calos MP: φC31 integrase confers genomic integration and long-term transgene expression in rat retina. Invest Ophthalmol Vis Sci (2005) 46(6):2140-2146.
    • (2005) Invest Ophthalmol Vis Sci , vol.46 , Issue.6 , pp. 2140-2146
    • Chalberg, T.W.1    Genise, H.L.2    Vollrath, D.3    Calos, M.P.4
  • 55
    • 0035945238 scopus 로고    scopus 로고
    • Directed evolution of a recombinase for improved genomic integration at a native human sequence
    • Sclimenti CR, Thyagarajan B, Calos MP: Directed evolution of a recombinase for improved genomic integration at a native human sequence. Nucleic Acids Res (2001) 29(24):5044-5051.
    • (2001) Nucleic Acids Res , vol.29 , Issue.24 , pp. 5044-5051
    • Sclimenti, C.R.1    Thyagarajan, B.2    Calos, M.P.3
  • 56
    • 17644425657 scopus 로고    scopus 로고
    • A direct comparison of two nonviral gene therapy vectors for somatic integration: In vivo evaluation of the bacteriophage integrase φC31 and the Sleeping Beauty transposase
    • Ehrhardt A, Xu H, Huang Z, Engler JA, Kay MA: A direct comparison of two nonviral gene therapy vectors for somatic integration: In vivo evaluation of the bacteriophage integrase φC31 and the Sleeping Beauty transposase. Mol Ther (2005) 11(5):695-706.
    • (2005) Mol Ther , vol.11 , Issue.5 , pp. 695-706
    • Ehrhardt, A.1    Xu, H.2    Huang, Z.3    Engler, J.A.4    Kay, M.A.5
  • 57
    • 29244445116 scopus 로고    scopus 로고
    • Post-translational modifications in proteins involved in blood coagulation
    • • This paper provides an excellent review of issues confronting successful expression of blood coagulation proteins
    • Hansson K, Stenflo J: Post-translational modifications in proteins involved in blood coagulation. J Thromb Haemost (2005) 3(12):2633-2648. • This paper provides an excellent review of issues confronting successful expression of blood coagulation proteins.
    • (2005) J Thromb Haemost , vol.3 , Issue.12 , pp. 2633-2648
    • Hansson, K.1    Stenflo, J.2
  • 58
    • 0034202522 scopus 로고    scopus 로고
    • 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
    • Miao CH, Ohashi K, Patijn GA, Meuse L, Ye X, Thompson AR, Kay MA: 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. Mol Ther (2000) 1(6):522-532.
    • (2000) Mol Ther , vol.1 , Issue.6 , pp. 522-532
    • Miao, C.H.1    Ohashi, K.2    Patijn, G.A.3    Meuse, L.4    Ye, X.5    Thompson, A.R.6    Kay, M.A.7
  • 59
    • 34247591462 scopus 로고    scopus 로고
    • The humoral response to human Factor VIII in hemophilia A mice
    • In a review of the immune reaction to Factor VIII in hemophilia A mice, this study suggested possible targets for reducing this response, •
    • Healey JF, Parker ET, Barrow RT, Langley TJ, Church WR, Lollar P: The humoral response to human Factor VIII in hemophilia A mice. J Thromb Haemost (2007) 5(3):512-519. • In a review of the immune reaction to Factor VIII in hemophilia A mice, this study suggested possible targets for reducing this response.
    • (2007) J Thromb Haemost , vol.5 , Issue.3 , pp. 512-519
    • Healey, J.F.1    Parker, E.T.2    Barrow, R.T.3    Langley, T.J.4    Church, W.R.5    Lollar, P.6
  • 61
    • 3242716186 scopus 로고    scopus 로고
    • Reduction of the inhibitory antibody response to human Factor VIII in hemophilia A mice by mutagenesis of the A2 domain B-cell epitope
    • Parker ET, Healey JF, Barrow RT, Craddock HN, Lollar P: Reduction of the inhibitory antibody response to human Factor VIII in hemophilia A mice by mutagenesis of the A2 domain B-cell epitope. Blood (2004) 104(3):704-710.
    • (2004) Blood , vol.104 , Issue.3 , pp. 704-710
    • Parker, E.T.1    Healey, J.F.2    Barrow, R.T.3    Craddock, H.N.4    Lollar, P.5
  • 62
    • 20444476217 scopus 로고    scopus 로고
    • Induction of tolerance to Factor VIII inhibitors by gene therapy with immunodominant A2 and C2 domains presented by B cells as Ig fusion proteins
    • Lei TC, Scott DW: Induction of tolerance to Factor VIII inhibitors by gene therapy with immunodominant A2 and C2 domains presented by B cells as Ig fusion proteins. Blood (2005) 105(12):4865-4870.
    • (2005) Blood , vol.105 , Issue.12 , pp. 4865-4870
    • Lei, T.C.1    Scott, D.W.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.