메뉴 건너뛰기




Volumn 26, Issue 6, 2015, Pages 211-220

Site-Directed Mutagenesis of Surface-Exposed Lysine Residues Leads to Improved Transduction by AAV2, But Not AAV8, Vectors in Murine Hepatocytes In Vivo

Author keywords

[No Author keywords available]

Indexed keywords

GLUTAMIC ACID; LYSINE; PARVOVIRUS VECTOR; PROTEASOME; UBIQUITIN; CAPSID PROTEIN;

EID: 84949666934     PISSN: 19466536     EISSN: 19466544     Source Type: Journal    
DOI: 10.1089/hgtb.2015.115     Document Type: Article
Times cited : (27)

References (39)
  • 1
    • 0029257496 scopus 로고
    • The cryptic life style of adeno-associated virus
    • Berns KI, Linden RM. The cryptic life style of adeno-associated virus. Bioessays 1995;17:237-245.
    • (1995) Bioessays , vol.17 , pp. 237-245
    • Berns, K.I.1    Linden, R.M.2
  • 2
    • 79954622209 scopus 로고    scopus 로고
    • Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges
    • Mingozzi F, High KA. Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges. Nat Rev Genet 2011;12:341-355.
    • (2011) Nat Rev Genet , vol.12 , pp. 341-355
    • Mingozzi, F.1    High, K.A.2
  • 3
    • 84883268937 scopus 로고    scopus 로고
    • Immune responses to AAV vectors: Overcoming barriers to successful gene therapy
    • Mingozzi F, High KA. Immune responses to AAV vectors: Overcoming barriers to successful gene therapy. Blood 2013;122:23-36.
    • (2013) Blood , vol.122 , pp. 23-36
    • Mingozzi, F.1    High, K.A.2
  • 4
    • 33644820684 scopus 로고    scopus 로고
    • Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    • Manno CS, Pierce GF, Arruda VR, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006;12:342-347.
    • (2006) Nat Med , vol.12 , pp. 342-347
    • Manno, C.S.1    Pierce, G.F.2    Arruda, V.R.3
  • 5
    • 34147098413 scopus 로고    scopus 로고
    • CD8(+) T-cell responses to adeno-associated virus capsid in humans
    • Mingozzi F, Maus MV, Hui DJ, et al. CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med 2007;13:419-422.
    • (2007) Nat Med , vol.13 , pp. 419-422
    • Mingozzi, F.1    Maus, M.V.2    Hui, D.J.3
  • 6
    • 67651006589 scopus 로고    scopus 로고
    • Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
    • Pien GC, Basner-Tschakarjan E, Hui DJ, et al. Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J Clin Invest 2009;119:1688-1695.
    • (2009) J Clin Invest , vol.119 , pp. 1688-1695
    • Pien, G.C.1    Basner-Tschakarjan, E.2    Hui, D.J.3
  • 7
    • 84855161388 scopus 로고    scopus 로고
    • Adenovirus-associated virus vectormediated gene transfer in hemophilia B
    • Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vectormediated gene transfer in hemophilia B. N Engl J Med 2011;365:2357-2365.
    • (2011) N Engl J Med , vol.365 , pp. 2357-2365
    • Nathwani, A.C.1    Tuddenham, E.G.2    Rangarajan, S.3
  • 8
    • 84911383748 scopus 로고    scopus 로고
    • Longterm safety and efficacy of factor IX gene therapy in hemophilia B
    • Nathwani AC, Reiss UM, Tuddenham EG, et al. Longterm safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014;371:1994-2004.
    • (2014) N Engl J Med , vol.371 , pp. 1994-2004
    • Nathwani, A.C.1    Reiss, U.M.2    Tuddenham, E.G.3
  • 9
    • 84897931068 scopus 로고    scopus 로고
    • Pristimerin enhances recombinant adeno-associated virus vector-mediated transgene expression in human cell lines in vitro and murine hepatocytes in vivo
    • Wang LN, Wang Y, Lu Y, et al. Pristimerin enhances recombinant adeno-associated virus vector-mediated transgene expression in human cell lines in vitro and murine hepatocytes in vivo. J Integr Med 2014;12:20-34.
    • (2014) J Integr Med , vol.12 , pp. 20-34
    • Wang, L.N.1    Wang, Y.2    Lu, Y.3
  • 10
    • 34250727017 scopus 로고    scopus 로고
    • A dual role of EGFR protein tyrosine kinase signaling in ubiquitination of AAV2 capsids and viral second-strand DNA synthesis
    • Zhong L, Zhao W, Wu J, et al. A dual role of EGFR protein tyrosine kinase signaling in ubiquitination of AAV2 capsids and viral second-strand DNA synthesis. Mol Ther 2007;15:1323-1330.
    • (2007) Mol Ther , vol.15 , pp. 1323-1330
    • Zhong, L.1    Zhao, W.2    Wu, J.3
  • 11
    • 54549096772 scopus 로고    scopus 로고
    • Tyrosinephosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression
    • Zhong L, Li B, Jayandharan G, et al. Tyrosinephosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression. Virology 2008;381:194-202.
    • (2008) Virology , vol.381 , pp. 194-202
    • Zhong, L.1    Li, B.2    Jayandharan, G.3
  • 12
    • 45549090635 scopus 로고    scopus 로고
    • Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
    • Zhong L, Li B, Mah CS, et al. Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci USA 2008;105:7827-7832.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 7827-7832
    • Zhong, L.1    Li, B.2    Mah, C.S.3
  • 13
    • 84875092627 scopus 로고    scopus 로고
    • Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: The final threshold?
    • Aslanidi GV, Rivers AE, Ortiz L, et al. Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: The final threshold? PLoS One 2013;8:e59142.
    • (2013) PLoS One , vol.8 , pp. e59142
    • Aslanidi, G.V.1    Rivers, A.E.2    Ortiz, L.3
  • 14
    • 84861189907 scopus 로고    scopus 로고
    • High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors
    • Aslanidi GV, Rivers AE, Ortiz L, et al. High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors. Vaccine 2012;30:3908-3917.
    • (2012) Vaccine , vol.30 , pp. 3908-3917
    • Aslanidi, G.V.1    Rivers, A.E.2    Ortiz, L.3
  • 16
    • 78650898983 scopus 로고    scopus 로고
    • High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines
    • Markusic DM, Herzog RW, Aslanidi GV, et al. High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines. Mol Ther 2010;18:2048-2056.
    • (2010) Mol Ther , vol.18 , pp. 2048-2056
    • Markusic, D.M.1    Herzog, R.W.2    Aslanidi, G.V.3
  • 17
    • 84055199890 scopus 로고    scopus 로고
    • Fate tracing of mature hepatocytes in mouse liver homeostasis and regeneration
    • Malato Y, Naqvi S, Schurmann N, et al. Fate tracing of mature hepatocytes in mouse liver homeostasis and regeneration. J Clin Invest 2011;121:4850-4860.
    • (2011) J Clin Invest , vol.121 , pp. 4850-4860
    • Malato, Y.1    Naqvi, S.2    Schurmann, N.3
  • 18
    • 10644289281 scopus 로고    scopus 로고
    • Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice
    • Nakai H, Fuess S, Storm TA, et al. Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J Virol 2005;79:214-224.
    • (2005) J Virol , vol.79 , pp. 214-224
    • Nakai, H.1    Fuess, S.2    Storm, T.A.3
  • 19
    • 84908295689 scopus 로고    scopus 로고
    • Low doses of ethanolic extract of Boldo (Peumus boldus) can ameliorate toxicity generated by cisplatin in normal liver cells of mice in vivo and in WRL-68 cells in vitro, but not in cancer cells in vivo or in vitro
    • Mondal J, Bishayee K, Panigrahi AK, et al. Low doses of ethanolic extract of Boldo (Peumus boldus) can ameliorate toxicity generated by cisplatin in normal liver cells of mice in vivo and in WRL-68 cells in vitro, but not in cancer cells in vivo or in vitro. J Integr Med 2014;12:425-438.
    • (2014) J Integr Med , vol.12 , pp. 425-438
    • Mondal, J.1    Bishayee, K.2    Panigrahi, A.K.3
  • 20
    • 84874759776 scopus 로고    scopus 로고
    • Endocytic processing of adeno-associated virus type 8 vectors for transduction of target cells
    • Liu Y, Joo KI, Wang P. Endocytic processing of adeno-associated virus type 8 vectors for transduction of target cells. Gene Ther 2013;20: 308-317.
    • (2013) Gene Ther , vol.20 , pp. 308-317
    • Liu, Y.1    Joo, K.I.2    Wang, P.3
  • 21
    • 84906671515 scopus 로고    scopus 로고
    • Enhancing gene delivery of adeno-associated viruses by cellpermeable peptides
    • Liu Y, Kim YJ, Ji M, et al. Enhancing gene delivery of adeno-associated viruses by cellpermeable peptides. Mol Ther Methods Clin Dev 2014;1:12.
    • (2014) Mol Ther Methods Clin Dev , vol.1 , pp. 12
    • Liu, Y.1    Kim, Y.J.2    Ji, M.3
  • 22
    • 84856215097 scopus 로고    scopus 로고
    • Altered LKB1/CREBregulated transcription co-activator (CRTC) signaling axis promotes esophageal cancer cell migration and invasion
    • Gu Y, Lin S, Li JL, et al. Altered LKB1/CREBregulated transcription co-activator (CRTC) signaling axis promotes esophageal cancer cell migration and invasion. Oncogene 2012;31:469-479.
    • (2012) Oncogene , vol.31 , pp. 469-479
    • Gu, Y.1    Lin, S.2    Li, J.L.3
  • 23
    • 84918780947 scopus 로고    scopus 로고
    • Cytotoxic genes from traditional Chinese medicine inhibit tumor growth both in vitro and in vivo
    • Zhang YH, Wang Y, Yusufali AH, et al. Cytotoxic genes from traditional Chinese medicine inhibit tumor growth both in vitro and in vivo. J Integr Med 2014;12:483-494.
    • (2014) J Integr Med , vol.12 , pp. 483-494
    • Zhang, Y.H.1    Wang, Y.2    Yusufali, A.H.3
  • 25
    • 58149182734 scopus 로고    scopus 로고
    • VIPER2: An enhanced and web API enabled relational database for structural virology
    • Carrillo-Trip M, Shepherd CM, Borelli IA, et al. VIPER2: an enhanced and web API enabled relational database for structural virology. Nucl Acids Res 2009;37:D436-D442.
    • (2009) Nucl Acids Res , vol.37 , pp. D436-D442
    • Carrillo-Trip, M.1    Shepherd, C.M.2    Borelli, I.A.3
  • 26
    • 84864192056 scopus 로고    scopus 로고
    • PyMod: Sequence similarity searches, multiple sequencestructure alignments, and homology modeling within PyMOL
    • Bramucci E, Paiardini A, Bossa F, et al. PyMod: Sequence similarity searches, multiple sequencestructure alignments, and homology modeling within PyMOL. BMC Bioinform 2012;13 Suppl 4:S2.
    • (2012) BMC Bioinform , vol.13 , pp. S2
    • Bramucci, E.1    Paiardini, A.2    Bossa, F.3
  • 27
    • 0036678455 scopus 로고    scopus 로고
    • The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy
    • Xie Q, Bu W, Bhatia S, et al. The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy. Proc Natl Acad Sci USA 2002;99: 10405-10410.
    • (2002) Proc Natl Acad Sci USA , vol.99 , pp. 10405-10410
    • Xie, Q.1    Bu, W.2    Bhatia, S.3
  • 28
    • 84878678218 scopus 로고    scopus 로고
    • Bioengineering of AAV2 capsid at specific serine, threonine, or lysine residues improves its transduction efficiency in vitro and in vivo
    • Gabriel N, Hareendran S, Sen D, et al. Bioengineering of AAV2 capsid at specific serine, threonine, or lysine residues improves its transduction efficiency in vitro and in vivo. Hum Gene Ther Methods 2013;24:80-93.
    • (2013) Hum Gene Ther Methods , vol.24 , pp. 80-93
    • Gabriel, N.1    Hareendran, S.2    Sen, D.3
  • 29
    • 44349170706 scopus 로고    scopus 로고
    • Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
    • Zincarelli C, Soltys S, Rengo G, et al. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 2008;16:1073-1080.
    • (2008) Mol Ther , vol.16 , pp. 1073-1080
    • Zincarelli, C.1    Soltys, S.2    Rengo, G.3
  • 30
    • 78650105132 scopus 로고    scopus 로고
    • Human hepatocyte growth factor receptor is a cellular coreceptor for adeno-associated virus serotype 3
    • Ling C, Lu Y, Kalsi JK, et al. Human hepatocyte growth factor receptor is a cellular coreceptor for adeno-associated virus serotype 3. Hum Gene Ther 2010;21:1741-1747.
    • (2010) Hum Gene Ther , vol.21 , pp. 1741-1747
    • Ling, C.1    Lu, Y.2    Kalsi, J.K.3
  • 31
    • 84892967754 scopus 로고    scopus 로고
    • Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo
    • Song L, Kauss MA, Kopin E, et al. Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo. Cytotherapy 2013;15:986-998.
    • (2013) Cytotherapy , vol.15 , pp. 986-998
    • Song, L.1    Kauss, M.A.2    Kopin, E.3
  • 32
    • 84878699709 scopus 로고    scopus 로고
    • Targeted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivo
    • Sen D, Gadkari RA, Sudha G, et al. Targeted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivo. Hum Gene Ther Methods 2013; 24:104-116.
    • (2013) Hum Gene Ther Methods , vol.24 , pp. 104-116
    • Sen, D.1    Gadkari, R.A.2    Sudha, G.3
  • 33
    • 84893407639 scopus 로고    scopus 로고
    • K137R mutation on adeno-associated viral capsids had minimal effect on enhancing gene delivery in vivo
    • Qiao C, Li C, Zhao C, et al. K137R mutation on adeno-associated viral capsids had minimal effect on enhancing gene delivery in vivo. Hum Gene Ther Methods 2014;25:33-39.
    • (2014) Hum Gene Ther Methods , vol.25 , pp. 33-39
    • Qiao, C.1    Li, C.2    Zhao, C.3
  • 34
    • 0033987571 scopus 로고    scopus 로고
    • Impaired intracellular trafficking of adeno-associated virus type 2 vectors limits efficient transduction of murine fibroblasts
    • Hansen J, Qing K, Kwon HJ, et al. Impaired intracellular trafficking of adeno-associated virus type 2 vectors limits efficient transduction of murine fibroblasts. J Virol 2000;74:992-996.
    • (2000) J Virol , vol.74 , pp. 992-996
    • Hansen, J.1    Qing, K.2    Kwon, H.J.3
  • 35
    • 0036171821 scopus 로고    scopus 로고
    • Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors
    • Yan Z, Zak R, Luxton GW, et al. Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors. J Virol 2002;76:2043-2053.
    • (2002) J Virol , vol.76 , pp. 2043-2053
    • Yan, Z.1    Zak, R.2    Luxton, G.W.3
  • 37
    • 30344450073 scopus 로고    scopus 로고
    • Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization
    • Lochrie MA, Tatsuno GP, Christie B, et al. Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization. J Virol 2006;80:821-834.
    • (2006) J Virol , vol.80 , pp. 821-834
    • Lochrie, M.A.1    Tatsuno, G.P.2    Christie, B.3
  • 38
    • 77957234673 scopus 로고    scopus 로고
    • An animal model of PDH deficiency using AAV8-siRNA vector-mediated knockdown of pyruvate dehydrogenase E1alpha
    • Ojano-Dirain C, Glushakova LG, Zhong L, et al. An animal model of PDH deficiency using AAV8-siRNA vector-mediated knockdown of pyruvate dehydrogenase E1alpha. Mol Genet Metab 2010; 101:183-191.
    • (2010) Mol Genet Metab , vol.101 , pp. 183-191
    • Ojano-Dirain, C.1    Glushakova, L.G.2    Zhong, L.3
  • 39
    • 74149089236 scopus 로고    scopus 로고
    • Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction
    • Finn JD, Hui D, Downey HD, et al. Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction. Mol Ther 2010;18:135-142.
    • (2010) Mol Ther , vol.18 , pp. 135-142
    • Finn, J.D.1    Hui, D.2    Downey, H.D.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.