메뉴 건너뛰기




Volumn 15, Issue 10, 2007, Pages 1856-1862

The enhancing effects of the light chain on heavy chain secretion in split delivery of factor VIII gene

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 8; HYBRID PROTEIN; INTEIN;

EID: 34548796475     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/sj.mt.6300268     Document Type: Article
Times cited : (47)

References (35)
  • 1
    • 0032697618 scopus 로고    scopus 로고
    • Biochemistry and physiology of blood coagulation
    • Mann, KG(1999). Biochemistry and physiology of blood coagulation. Thromb Haemost 82: 165-174.
    • (1999) Thromb Haemost , vol.82 , pp. 165-174
    • Mann, K.G.1
  • 2
    • 0033200188 scopus 로고    scopus 로고
    • Advances toward gene therapy for hemophilia at the millennium
    • Kaufman, RJ (1999). Advances toward gene therapy for hemophilia at the millennium. Hum Gene Ther 10, 2091-2107.
    • (1999) Hum Gene Ther , vol.10 , pp. 2091-2107
    • Kaufman, R.J.1
  • 3
    • 2342542505 scopus 로고    scopus 로고
    • Coagulation factors with improved properties for hemophilia gene therapy
    • Pipe, SW (2004). Coagulation factors with improved properties for hemophilia gene therapy. Semin Thromb Hemost 30: 227-237.
    • (2004) Semin Thromb Hemost , vol.30 , pp. 227-237
    • Pipe, S.W.1
  • 4
    • 2342656515 scopus 로고    scopus 로고
    • Preclinical gene therapy studies for hemophilia using adeno-associated virus (AAV) vectors
    • Couto, LB (2004). Preclinical gene therapy studies for hemophilia using adeno-associated virus (AAV) vectors. Semin Thromb Hemost 30 161-171.
    • (2004) Semin Thromb Hemost , vol.30 , pp. 161-171
    • Couto, L.B.1
  • 5
    • 0033621122 scopus 로고    scopus 로고
    • Gene therapy for the hemophilias [comment]
    • Kay, MA and High, K (1999). Gene therapy for the hemophilias [comment]. Proc Natl Acad Sd USA 96: 9973-9975.
    • (1999) Proc Natl Acad Sd USA , vol.96 , pp. 9973-9975
    • Kay, M.A.1    High, K.2
  • 6
    • 2342457825 scopus 로고    scopus 로고
    • Clinical gene transfer studies for hemophilia B
    • High, KA (2004). Clinical gene transfer studies for hemophilia B. Semin Thromb Hemost 30: 257-267.
    • (2004) Semin Thromb Hemost , vol.30 , pp. 257-267
    • High, K.A.1
  • 7
    • 33745606715 scopus 로고    scopus 로고
    • Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs
    • Jiang, H, Lillicrap, D, Patarroyo-White, S, Liu, T, Qian, X, Scallan, CD et al. (2006). Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs. Blood 108: 107-115.
    • (2006) Blood , vol.108 , pp. 107-115
    • Jiang, H.1    Lillicrap, D.2    Patarroyo-White, S.3    Liu, T.4    Qian, X.5    Scallan, C.D.6
  • 8
    • 0141450407 scopus 로고    scopus 로고
    • A single adeno-associated virus (AAV)-murine factor VIII vector partially corrects the hemophilia A phenotype
    • Sarkar, R, Xiao, W and Kazazian, HH Jr. (2003). A single adeno-associated virus (AAV)-murine factor VIII vector partially corrects the hemophilia A phenotype. J Thromb Haemost 1: 220-226.
    • (2003) J Thromb Haemost , vol.1 , pp. 220-226
    • Sarkar, R.1    Xiao, W.2    Kazazian Jr., H.H.3
  • 9
    • 10744230709 scopus 로고    scopus 로고
    • Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII
    • Scallan, CD, Liu, T, Parker, AE, Patarrovo-White, SL, Chen, H, Jiang, H et al. (2003). Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII. Blood 102: 3919-3926.
    • (2003) Blood , vol.102 , pp. 3919-3926
    • Scallan, C.D.1    Liu, T.2    Parker, A.E.3    Patarrovo-White, S.L.4    Chen, H.5    Jiang, H.6
  • 10
    • 0033607305 scopus 로고    scopus 로고
    • Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein
    • Burton, M, Nakai, H, Colosi, P, Cunningham, J, Mitchell, R and Couto, L (1999). Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein. Proc Natl Acad Sci USA 96: 12725-12730.
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 12725-12730
    • Burton, M.1    Nakai, H.2    Colosi, P.3    Cunningham, J.4    Mitchell, R.5    Couto, L.6
  • 11
    • 0037255184 scopus 로고    scopus 로고
    • Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice
    • Mah, C, Sarkar, R, Zolotukhin, I, Schleissing, M, Xiao, X, Kazazian, HH et al. (2003). Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice. Hum Gene Ther 14 143-152.
    • (2003) Hum Gene Ther , vol.14 , pp. 143-152
    • Mah, C.1    Sarkar, R.2    Zolotukhin, I.3    Schleissing, M.4    Xiao, X.5    Kazazian, H.H.6
  • 12
    • 0024965760 scopus 로고
    • Genetic engineering of factor VIII
    • Kaufman, RJ (1989). Genetic engineering of factor VIII. Nature 342: 207-208.
    • (1989) Nature , vol.342 , pp. 207-208
    • Kaufman, R.J.1
  • 16
    • 0024404910 scopus 로고
    • Increased synthesis of secreted proteins induces expression of glucose-regulated proteins in butyrate-treated Chinese hamster ovary cells
    • Domer, AJ, Wasley, LC and Kaufman, RJ (1989). Increased synthesis of secreted proteins induces expression of glucose-regulated proteins in butyrate-treated Chinese hamster ovary cells. J Biol Chem 264 20602-20607.
    • (1989) J Biol Chem , vol.264 , pp. 20602-20607
    • Domer, A.J.1    Wasley, L.C.2    Kaufman, R.J.3
  • 17
    • 0031876273 scopus 로고    scopus 로고
    • Can we improve on nature? "Super molecules" of factor VIII
    • Kaufman, RJ and Pipe, SW (1998). Can we improve on nature? "Super molecules" of factor VIII. Haemophilia 4: 370-379.
    • (1998) Haemophilia , vol.4 , pp. 370-379
    • Kaufman, R.J.1    Pipe, S.W.2
  • 18
    • 0022760260 scopus 로고
    • A large region (approximately equal to 95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity
    • Toole, JJ, Pittman, DD, Orr, EC, Murtha, P, Wasley, LC and Kaulman, RJ (1986). A large region (approximately equal to 95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity, Proc Natl Acad Sci USA 83: 5939-5942.
    • (1986) Proc Natl Acad Sci USA , vol.83 , pp. 5939-5942
    • Toole, J.J.1    Pittman, D.D.2    Orr, E.C.3    Murtha, P.4    Wasley, L.C.5    Kaulman, R.J.6
  • 19
    • 0028241866 scopus 로고
    • Post-translational requirements for functional factor V and factor VIII secretion in mammalian cells
    • Pittman, DD, Tomkinson, KN and Kaufman, RJ (1994). Post-translational requirements for functional factor V and factor VIII secretion in mammalian cells. J Biol Chem 269: 17329-17337.
    • (1994) J Biol Chem , vol.269 , pp. 17329-17337
    • Pittman, D.D.1    Tomkinson, K.N.2    Kaufman, R.J.3
  • 20
    • 3042741124 scopus 로고    scopus 로고
    • The ins and outs of gene expression control
    • Perler, FB (2004). The ins and outs of gene expression control. Nat Biotechnol 22: 824-826.
    • (2004) Nat Biotechnol , vol.22 , pp. 824-826
    • Perler, F.B.1
  • 21
    • 0032483013 scopus 로고    scopus 로고
    • Protein trans-splicing by a spilt intein encoded in a split DnaE gene of Synechocystis sp. PCC6803
    • Wu, H, Hu, Z and Liu, XQ (1998). Protein trans-splicing by a spilt intein encoded in a split DnaE gene of Synechocystis sp. PCC6803. Proc Natl Acad Sci USA 95: 9226-9231.
    • (1998) Proc Natl Acad Sci USA , vol.95 , pp. 9226-9231
    • Wu, H.1    Hu, Z.2    Liu, X.Q.3
  • 22
    • 0030753081 scopus 로고    scopus 로고
    • A DnaB intein in Rhodothermus marinus: Indication of recent intein homing across remotely related organisms
    • Liu, XQ and Hu, Z (1997). A DnaB intein in Rhodothermus marinus: Indication of recent intein homing across remotely related organisms. Proc Natl Acad Sci USA 94: 7851-7856.
    • (1997) Proc Natl Acad Sci USA , vol.94 , pp. 7851-7856
    • Liu, X.Q.1    Hu, Z.2
  • 23
    • 18144439565 scopus 로고    scopus 로고
    • Protein trans-splicing and functional mini-inteins of a cyanobacterial dnaB intein
    • Wu, H, Xu, MQ and Liu, XQ (1998). Protein trans-splicing and functional mini-inteins of a cyanobacterial dnaB intein. Biochim Biophys Acta 1387:422-432.
    • (1998) Biochim Biophys Acta , vol.1387 , pp. 422-432
    • Wu, H.1    Xu, M.Q.2    Liu, X.Q.3
  • 24
    • 0037325163 scopus 로고    scopus 로고
    • Structure and function of the factor VIII gene and protein
    • Thompson, AR (2003). Structure and function of the factor VIII gene and protein. Semin Thromb Hemost 29:11-22.
    • (2003) Semin Thromb Hemost , vol.29 , pp. 11-22
    • Thompson, A.R.1
  • 25
    • 32044453080 scopus 로고    scopus 로고
    • Endoplasmic reticulum stress activates cleavage of CREBH to incluce a systemic inflammatory response
    • Zhang, K, Shen, X, Wu, J, Sakaki, K, Saunders, T, Rutkowski, DT et al. (2006). Endoplasmic reticulum stress activates cleavage of CREBH to incluce a systemic inflammatory response. Cell 124: 587-599.
    • (2006) Cell , vol.124 , pp. 587-599
    • Zhang, K.1    Shen, X.2    Wu, J.3    Sakaki, K.4    Saunders, T.5    Rutkowski, D.T.6
  • 27
    • 28244484911 scopus 로고    scopus 로고
    • The promise and challenges of bioengineered recombinant clotting factors
    • Pipe, SW (2005). The promise and challenges of bioengineered recombinant clotting factors. J Thromb Haemost 3: 1692-1701.
    • (2005) J Thromb Haemost , vol.3 , pp. 1692-1701
    • Pipe, S.W.1
  • 28
    • 0029840409 scopus 로고    scopus 로고
    • Factor VIII C2 domain missense mutations exhibit defective trafficking of biologically functional proteins
    • Pipe, SW and Kaufman, RJ (1996). Factor VIII C2 domain missense mutations exhibit defective trafficking of biologically functional proteins. J Biol Chem 271:25671-25676.
    • (1996) J Biol Chem , vol.271 , pp. 25671-25676
    • Pipe, S.W.1    Kaufman, R.J.2
  • 29
    • 0031804517 scopus 로고    scopus 로고
    • Kemball-Cook, G, Tuddenham, EG and Wacey, AJ 1998, The factor VIII Structure and Mutation Resource Site: HAMSTeRS version 4. Nucliec Acids Res 26: 216-219
    • Kemball-Cook, G, Tuddenham, EG and Wacey, AJ (1998). The factor VIII Structure and Mutation Resource Site: HAMSTeRS version 4. Nucliec Acids Res 26: 216-219.
  • 30
    • 0031841277 scopus 로고    scopus 로고
    • Haemophilia B: Database of point mutations and short additions and delletions, eighth edition
    • Giannelli, F, Green, PM, Sommer, SS, Poon, M, Ludwiq, M, Schwaab, R et al. (1998). Haemophilia B: Database of point mutations and short additions and delletions - eighth edition. Nucleic Acids Res 26 265-268.
    • (1998) Nucleic Acids Res , vol.26 , pp. 265-268
    • Giannelli, F.1    Green, P.M.2    Sommer, S.S.3    Poon, M.4    Ludwiq, M.5    Schwaab, R.6
  • 31
    • 0348038748 scopus 로고    scopus 로고
    • Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo
    • Wang, Z, Ma, HI, Li, J, Sun, L, Zhang, J and Xiao, X (2003). Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther 10: 2105-2111.
    • (2003) Gene Ther , vol.10 , pp. 2105-2111
    • Wang, Z.1    Ma, H.I.2    Li, J.3    Sun, L.4    Zhang, J.5    Xiao, X.6
  • 32
    • 0029843596 scopus 로고    scopus 로고
    • Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
    • Xiao, X, Li, J and Samulski, RJ (1996). Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 70: 8098-8108.
    • (1996) J Virol , vol.70 , pp. 8098-8108
    • Xiao, X.1    Li, J.2    Samulski, R.J.3
  • 33
    • 0842264039 scopus 로고    scopus 로고
    • Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype
    • Sarkar, R, Tetreault, R, Gao, G, Wang, L, Bell, P, Chandler, R et al. (2004). Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. Blood 103: 1253-1260.
    • (2004) Blood , vol.103 , pp. 1253-1260
    • Sarkar, R.1    Tetreault, R.2    Gao, G.3    Wang, L.4    Bell, P.5    Chandler, R.6
  • 34
    • 0036369564 scopus 로고    scopus 로고
    • Hydrodynamics-based transfection: Simple and efficient method for introducing and expressing transgenes in animals by intravenous injection of DNA
    • Song, YK, Liu, F, Zhang, G and Liu, D (2002). Hydrodynamics-based transfection: Simple and efficient method for introducing and expressing transgenes in animals by intravenous injection of DNA. Methods Enzymol 346:92-105.
    • (2002) Methods Enzymol , vol.346 , pp. 92-105
    • Song, Y.K.1    Liu, F.2    Zhang, G.3    Liu, D.4
  • 35
    • 0032805251 scopus 로고    scopus 로고
    • Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA
    • Liu, F, Song, Y and Liu, D (1999). Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther 6 1258-1266.
    • (1999) Gene Ther , vol.6 , pp. 1258-1266
    • Liu, F.1    Song, Y.2    Liu, D.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.