메뉴 건너뛰기




Volumn 53, Issue 6, 2010, Pages 683-689

Protein trans-splicing based dual-vector delivery of the coagulation factor VIII gene

Author keywords

Coagulation factor VIII; Dual vector gene delivery; Intein; Protein trans splicing

Indexed keywords

B DOMAIN DELETED FACTOR VIII; B-DOMAIN-DELETED FACTOR VIII; BLOOD CLOTTING FACTOR 8; PEPTIDE FRAGMENT; PRIMER DNA;

EID: 77954299403     PISSN: 16747305     EISSN: None     Source Type: Journal    
DOI: 10.1007/s11427-010-4011-7     Document Type: Article
Times cited : (27)

References (23)
  • 1
    • 33748551323 scopus 로고    scopus 로고
    • Protein splicing in cis and in trans
    • Saleh L, Perler F B. Protein splicing in cis and in trans. Chem Rec, 2006, 6: 183-193.
    • (2006) Chem Rec , vol.6 , pp. 183-193
    • Saleh, L.1    Perler, F.B.2
  • 2
    • 18144439565 scopus 로고    scopus 로고
    • Protein trans-splicing and functional mini-inteins of a cyanobacterial dnaB intein
    • Wu H, Xu M Q, Liu X Q. Protein trans-splicing and functional mini-inteins of a cyanobacterial dnaB intein. Biochim Biophys Acta, 1998, 1387: 422-432.
    • (1998) Biochim Biophys Acta , vol.1387 , pp. 422-432
    • Wu, H.1    Xu, M.Q.2    Liu, X.Q.3
  • 3
    • 0032483013 scopus 로고    scopus 로고
    • Protein trans-splicing by a split intein encoded in a split DnaE gene of Synechocystis sp
    • Wu H, Hu Z, Liu X Q. Protein trans-splicing by a split intein encoded in a split DnaE gene of Synechocystis sp. PCC6803. Proc Natl Acad Sci USA, 1998, 95: 9226-9231.
    • (1998) PCC6803. Proc Natl Acad Sci USA , vol.95 , pp. 9226-9231
    • Wu, H.1    Hu, Z.2    Liu, X.Q.3
  • 4
    • 0029838842 scopus 로고    scopus 로고
    • The mechanism of protein splicing and its modulation by mutation
    • Xu M Q, Perler F B. The mechanism of protein splicing and its modulation by mutation. EMBO J, 1996, 15: 5146-5153.
    • (1996) EMBO J , vol.15 , pp. 5146-5153
    • Xu, M.Q.1    Perler, F.B.2
  • 5
    • 0032958487 scopus 로고    scopus 로고
    • Human factor VIII can be packaged and functionally expressed in an adeno-associated virus background: Applicability to haemophilia A gene therapy
    • Gnatenko D V, Saneko E L, Jesty J, et al. Human factor VIII can be packaged and functionally expressed in an adeno-associated virus background: applicability to haemophilia A gene therapy. Br J Haematol, 1999, 104: 27-36.
    • (1999) Br J Haematol , vol.104 , pp. 27-36
    • Gnatenko, D.V.1    Saneko, E.L.2    Jesty, J.3
  • 6
    • 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, et al. Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype. Blood, 2004, 103: 1253-1260.
    • (2004) Blood , vol.103 , pp. 1253-1260
    • Sarkar, R.1    Tetreault, R.2    Gao, G.3
  • 7
    • 33745991799 scopus 로고    scopus 로고
    • Intein-mediated protein purification of fusion proteins expressed under high-cell density conditions in E. coli
    • Sharma S S, Chong S, Harcum S W. Intein-mediated protein purification of fusion proteins expressed under high-cell density conditions in E. coli. J Biotechnol, 2006, 125: 48-56.
    • (2006) J Biotechnol , vol.125 , pp. 48-56
    • Sharma, S.S.1    Chong, S.2    Harcum, S.W.3
  • 8
    • 0037040880 scopus 로고    scopus 로고
    • In vivo protein cyclization promoted by a circularly permuted Synechocystis sp. PCC6803 DnaB mini-intein
    • Williams N K, Prosselkov P, Liepinsh E, et al. In vivo protein cyclization promoted by a circularly permuted Synechocystis sp. PCC6803 DnaB mini-intein. J Biol Chem, 2002, 277: 7790-7798.
    • (2002) J Biol Chem , vol.277 , pp. 7790-7798
    • Williams, N.K.1    Prosselkov, P.2    Liepinsh, E.3
  • 9
    • 54449093009 scopus 로고    scopus 로고
    • Interaction studies and alanine scanning analysis of a semi-synthetic split intein reveal thiazoline ring formation from an intermediate of the protein splicing reaction
    • Ludwig C, Schwarzer D, Mootz H D. Interaction studies and alanine scanning analysis of a semi-synthetic split intein reveal thiazoline ring formation from an intermediate of the protein splicing reaction. J Biol Chem, 2008, 283: 25264-25272.
    • (2008) J Biol Chem , vol.283 , pp. 25264-25272
    • Ludwig, C.1    Schwarzer, D.2    Mootz, H.D.3
  • 10
    • 0033200188 scopus 로고    scopus 로고
    • Advances toward gene therapy for hemophilia at the millennium
    • Kaufman R J. Advances toward gene therapy for hemophilia at the millennium. Hum Gene Ther, 1999, 10: 2091-2107.
    • (1999) Hum Gene Ther , vol.10 , pp. 2091-2107
    • Kaufman, R.J.1
  • 11
    • 38949118647 scopus 로고    scopus 로고
    • Gene therapy for haemophilia
    • Murphy S L, High K A. Gene therapy for haemophilia. Br J Haematol, 2008, 140: 479-487.
    • (2008) Br J Haematol , vol.140 , pp. 479-487
    • Murphy, S.L.1    High, K.A.2
  • 12
    • 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 C D, Liu T, Parker A E, et al. Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII. Blood, 2003, 102: 3919-3926.
    • (2003) Blood , vol.102 , pp. 3919-3926
    • Scallan, C.D.1    Liu, T.2    Parker, A.E.3
  • 13
    • 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, et al. Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein. Proc Natl Acad Sci USA, 1999, 96: 12725-12730.
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 12725-12730
    • Burton, M.1    Nakai, H.2    Colosi, P.3
  • 14
    • 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, et al. Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice. Hum Gene Ther, 2003, 14: 143-152.
    • (2003) Hum Gene Ther , vol.14 , pp. 143-152
    • Mah, C.1    Sarkar, R.2    Zolotukhin, I.3
  • 15
    • 32044453080 scopus 로고    scopus 로고
    • Endoplasmic reticulum stress activates cleavage of CREBH to induce a systemic inflammatory response
    • Zhang K, Shen X, Wu J, et al. Endoplasmic reticulum stress activates cleavage of CREBH to induce a systemic inflammatory response. Cell, 2006, 124: 587-599.
    • (2006) Cell , vol.124 , pp. 587-599
    • Zhang, K.1    Shen, X.2    Wu, J.3
  • 16
    • 0035988996 scopus 로고    scopus 로고
    • Expression of human factor VIII by splicing between dimerized AAV vectors
    • Chao H, Sun L, Bruce A, et al. Expression of human factor VIII by splicing between dimerized AAV vectors. Mol Ther, 2002, 5: 716-722.
    • (2002) Mol Ther , vol.5 , pp. 716-722
    • Chao, H.1    Sun, L.2    Bruce, A.3
  • 17
    • 0034121579 scopus 로고    scopus 로고
    • Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization
    • Sun L, Li J, Xiao X. Overcoming adeno-associated virus vector size limitation through viral DNA heterodimerization. Nat Med, 2000, 6: 599-602.
    • (2000) Nat Med , vol.6 , pp. 599-602
    • Sun, L.1    Li, J.2    Xiao, X.3
  • 18
    • 0034612248 scopus 로고    scopus 로고
    • Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy
    • Yan Z, Zhang Y, Duan D, et al. Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy. Proc Natl Acad Sci USA, 2000, 97: 6716-6721.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 6716-6721
    • Yan, Z.1    Zhang, Y.2    Duan, D.3
  • 19
    • 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, Kazazian H H Jr. A single adeno-associated virus (AAV)-murine factor VIII vector partially corrects the hemophilia A phenotype. J Thromb Haemost, 2003, 1: 220-226.
    • (2003) J Thromb Haemost , vol.1 , pp. 220-226
    • Sarkar, R.1    Xiao, W.2    Kazazian Jr., H.H.3
  • 20
    • 33748780913 scopus 로고    scopus 로고
    • Phenotype correction of hemophilia A mice with adeno-associated virus vectors carrying the B domain-deleted canine factor VIII gene
    • Ishiwata A, Mimuro J, Kashiwakura Y, et al. Phenotype correction of hemophilia A mice with adeno-associated virus vectors carrying the B domain-deleted canine factor VIII gene. Thromb Res, 2006, 118: 627-635.
    • (2006) Thromb Res , vol.118 , pp. 627-635
    • Ishiwata, A.1    Mimuro, J.2    Kashiwakura, Y.3
  • 21
    • 0141456083 scopus 로고    scopus 로고
    • Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector
    • Scallan C D, Lillicrap D, Jiang H, et al. Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector. Blood, 2003, 102: 2031-2037.
    • (2003) Blood , vol.102 , pp. 2031-2037
    • Scallan, C.D.1    Lillicrap, D.2    Jiang, H.3
  • 22
    • 34548796475 scopus 로고    scopus 로고
    • The enhancing effects of the light chain on heavy chain secretion in split delivery of factor VIII gene
    • Chen L X, Zhu F X, Li J, et al. The enhancing effects of the light chain on heavy chain secretion in split delivery of factor VIII gene. Mol Ther, 2007, 15: 1856-1862.
    • (2007) Mol Ther , vol.15 , pp. 1856-1862
    • Chen, L.X.1    Zhu, F.X.2    Li, J.3
  • 23
    • 77954275671 scopus 로고    scopus 로고
    • Split-intein mediated high efficient cellular fusion of transcription factor
    • Gao Y J, Zhao C K, Yin Q, et al. Split-intein mediated high efficient cellular fusion of transcription factor. Sci China Ser C-Life Sci, 2008, 38: 714-721.
    • (2008) Sci China Ser C-Life Sci , vol.38 , pp. 714-721
    • Gao, Y.J.1    Zhao, C.K.2    Yin, Q.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.