메뉴 건너뛰기




Volumn 8, Issue , 2017, Pages 184-197

Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS

Author keywords

AAV; capsid; CNS; mutant

Indexed keywords

GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; HEPARAN SULFATE; OLIGODENDROCYTE TRANSCRIPTION FACTOR 2; THREONINE; TYROSINE; VIRUS VECTOR;

EID: 85029280741     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1016/j.omtn.2017.06.011     Document Type: Article
Times cited : (51)

References (57)
  • 2
    • 77953722282 scopus 로고    scopus 로고
    • Development of gene therapy for neurological disorders
    • Manfredsson, F.P., Mandel, R.J., Development of gene therapy for neurological disorders. Discov. Med. 9 (2010), 204–211.
    • (2010) Discov. Med. , vol.9 , pp. 204-211
    • Manfredsson, F.P.1    Mandel, R.J.2
  • 3
    • 84987600814 scopus 로고    scopus 로고
    • Long-term safety of patients with Parkinson's disease receiving rAAV2-neurturin (CERE-120) gene transfer
    • Marks, W.J. Jr., Baumann, T.L., Bartus, R.T., Long-term safety of patients with Parkinson's disease receiving rAAV2-neurturin (CERE-120) gene transfer. Hum. Gene Ther. 27 (2016), 522–527.
    • (2016) Hum. Gene Ther. , vol.27 , pp. 522-527
    • Marks, W.J.1    Baumann, T.L.2    Bartus, R.T.3
  • 7
    • 32344438754 scopus 로고    scopus 로고
    • Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
    • Maheshri, N., Koerber, J.T., Kaspar, B.K., Schaffer, D.V., Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat. Biotechnol. 24 (2006), 198–204.
    • (2006) Nat. Biotechnol. , vol.24 , pp. 198-204
    • Maheshri, N.1    Koerber, J.T.2    Kaspar, B.K.3    Schaffer, D.V.4
  • 8
    • 44949131860 scopus 로고    scopus 로고
    • In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses
    • Grimm, D., Lee, J.S., Wang, L., Desai, T., Akache, B., Storm, T.A., Kay, M.A., In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J. Virol. 82 (2008), 5887–5911.
    • (2008) J. Virol. , vol.82 , pp. 5887-5911
    • Grimm, D.1    Lee, J.S.2    Wang, L.3    Desai, T.4    Akache, B.5    Storm, T.A.6    Kay, M.A.7
  • 12
    • 84897016759 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus utilizes host cell nuclear import machinery to enter the nucleus
    • Nicolson, S.C., Samulski, R.J., Recombinant adeno-associated virus utilizes host cell nuclear import machinery to enter the nucleus. J. Virol. 88 (2014), 4132–4144.
    • (2014) J. Virol. , vol.88 , pp. 4132-4144
    • Nicolson, S.C.1    Samulski, R.J.2
  • 13
    • 84856516466 scopus 로고    scopus 로고
    • Quantitative 3D tracing of gene-delivery viral vectors in human cells and animal tissues
    • Xiao, P.J., Li, C., Neumann, A., Samulski, R.J., Quantitative 3D tracing of gene-delivery viral vectors in human cells and animal tissues. Mol. Ther. 20 (2012), 317–328.
    • (2012) Mol. Ther. , vol.20 , pp. 317-328
    • Xiao, P.J.1    Li, C.2    Neumann, A.3    Samulski, R.J.4
  • 14
    • 84869015419 scopus 로고    scopus 로고
    • Cytoplasmic trafficking, endosomal escape, and perinuclear accumulation of adeno-associated virus type 2 particles are facilitated by microtubule network
    • Xiao, P.J., Samulski, R.J., Cytoplasmic trafficking, endosomal escape, and perinuclear accumulation of adeno-associated virus type 2 particles are facilitated by microtubule network. J. Virol. 86 (2012), 10462–10473.
    • (2012) J. Virol. , vol.86 , pp. 10462-10473
    • Xiao, P.J.1    Samulski, R.J.2
  • 16
    • 84893847671 scopus 로고    scopus 로고
    • Rationally designed capsid and transgene cassette of AAV6 vectors for dendritic cell-based cancer immunotherapy
    • Pandya, J., Ortiz, L., Ling, C., Rivers, A.E., Aslanidi, G., Rationally designed capsid and transgene cassette of AAV6 vectors for dendritic cell-based cancer immunotherapy. Immunol. Cell Biol. 92 (2014), 116–123.
    • (2014) Immunol. Cell Biol. , vol.92 , pp. 116-123
    • Pandya, J.1    Ortiz, L.2    Ling, C.3    Rivers, A.E.4    Aslanidi, G.5
  • 17
    • 0031906147 scopus 로고    scopus 로고
    • Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions
    • Summerford, C., Samulski, R.J., Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J. Virol. 72 (1998), 1438–1445.
    • (1998) J. Virol. , vol.72 , pp. 1438-1445
    • Summerford, C.1    Samulski, R.J.2
  • 19
    • 33745776920 scopus 로고    scopus 로고
    • Heparan sulfate proteoglycan binding properties of adeno-associated virus retargeting mutants and consequences for their in vivo tropism
    • Perabo, L., Goldnau, D., White, K., Endell, J., Boucas, J., Humme, S., Work, L.M., Janicki, H., Hallek, M., Baker, A.H., Büning, H., Heparan sulfate proteoglycan binding properties of adeno-associated virus retargeting mutants and consequences for their in vivo tropism. J. Virol. 80 (2006), 7265–7269.
    • (2006) J. Virol. , vol.80 , pp. 7265-7269
    • Perabo, L.1    Goldnau, D.2    White, K.3    Endell, J.4    Boucas, J.5    Humme, S.6    Work, L.M.7    Janicki, H.8    Hallek, M.9    Baker, A.H.10    Büning, H.11
  • 20
    • 33645456779 scopus 로고    scopus 로고
    • Insertional mutagenesis at positions 520 and 584 of adeno-associated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors with eliminated heparin- binding ability and introduced novel tropism
    • Shi, X., Fang, G., Shi, W., Bartlett, J.S., Insertional mutagenesis at positions 520 and 584 of adeno-associated virus type 2 (AAV2) capsid gene and generation of AAV2 vectors with eliminated heparin- binding ability and introduced novel tropism. Hum. Gene Ther. 17 (2006), 353–361.
    • (2006) Hum. Gene Ther. , vol.17 , pp. 353-361
    • Shi, X.1    Fang, G.2    Shi, W.3    Bartlett, J.S.4
  • 23
    • 34547118339 scopus 로고    scopus 로고
    • Time course of transgene expression after intrastriatal pseudotyped rAAV2/1, rAAV2/2, rAAV2/5, and rAAV2/8 transduction in the rat
    • Reimsnider, S., Manfredsson, F.P., Muzyczka, N., Mandel, R.J., Time course of transgene expression after intrastriatal pseudotyped rAAV2/1, rAAV2/2, rAAV2/5, and rAAV2/8 transduction in the rat. Mol. Ther. 15 (2007), 1504–1511.
    • (2007) Mol. Ther. , vol.15 , pp. 1504-1511
    • Reimsnider, S.1    Manfredsson, F.P.2    Muzyczka, N.3    Mandel, R.J.4
  • 24
    • 84884691211 scopus 로고    scopus 로고
    • Analysis of transduction efficiency, tropism and axonal transport of AAV serotypes 1, 2, 5, 6, 8 and 9 in the mouse brain
    • Aschauer, D.F., Kreuz, S., Rumpel, S., Analysis of transduction efficiency, tropism and axonal transport of AAV serotypes 1, 2, 5, 6, 8 and 9 in the mouse brain. PLoS ONE, 8, 2013, e76310.
    • (2013) PLoS ONE , vol.8 , pp. e76310
    • Aschauer, D.F.1    Kreuz, S.2    Rumpel, S.3
  • 26
    • 33846312858 scopus 로고    scopus 로고
    • Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS
    • Liu, G., Chen, Y.H., He, X., Martins, I., Heth, J.A., Chiorini, J.A., Davidson, B.L., Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS. Mol. Ther. 15 (2007), 242–247.
    • (2007) Mol. Ther. , vol.15 , pp. 242-247
    • Liu, G.1    Chen, Y.H.2    He, X.3    Martins, I.4    Heth, J.A.5    Chiorini, J.A.6    Davidson, B.L.7
  • 27
    • 0034968125 scopus 로고    scopus 로고
    • Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer
    • Hildinger, M., Auricchio, A., Gao, G., Wang, L., Chirmule, N., Wilson, J.M., Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer. J. Virol. 75 (2001), 6199–6203.
    • (2001) J. Virol. , vol.75 , pp. 6199-6203
    • Hildinger, M.1    Auricchio, A.2    Gao, G.3    Wang, L.4    Chirmule, N.5    Wilson, J.M.6
  • 28
    • 84859440283 scopus 로고    scopus 로고
    • The AAV vector toolkit: poised at the clinical crossroads
    • Asokan, A., Schaffer, D.V., Samulski, R.J., The AAV vector toolkit: poised at the clinical crossroads. Mol. Ther. 20 (2012), 699–708.
    • (2012) Mol. Ther. , vol.20 , pp. 699-708
    • Asokan, A.1    Schaffer, D.V.2    Samulski, R.J.3
  • 29
    • 0033557611 scopus 로고    scopus 로고
    • Oligodendrocyte-specific gene expression in mouse brain: use of a myelin-forming cell type-specific promoter in an adeno-associated virus
    • Chen, H., McCarty, D.M., Bruce, A.T., Suzuki, K., Oligodendrocyte-specific gene expression in mouse brain: use of a myelin-forming cell type-specific promoter in an adeno-associated virus. J. Neurosci. Res. 55 (1999), 504–513.
    • (1999) J. Neurosci. Res. , vol.55 , pp. 504-513
    • Chen, H.1    McCarty, D.M.2    Bruce, A.T.3    Suzuki, K.4
  • 30
    • 2442646815 scopus 로고    scopus 로고
    • Adeno-associated viral vector-mediated ApoE expression in Alzheimer's disease mice: low CNS immune response, long-term expression, and astrocyte specificity
    • Feng, X., Eide, F.F., Jiang, H., Reder, A.T., Adeno-associated viral vector-mediated ApoE expression in Alzheimer's disease mice: low CNS immune response, long-term expression, and astrocyte specificity. Front. Biosci. 9 (2004), 1540–1546.
    • (2004) Front. Biosci. , vol.9 , pp. 1540-1546
    • Feng, X.1    Eide, F.F.2    Jiang, H.3    Reder, A.T.4
  • 32
    • 79955602297 scopus 로고    scopus 로고
    • Quantifying transduction efficiencies of unmodified and tyrosine capsid mutant AAV vectors in vitro using two ocular cell lines
    • Ryals, R.C., Boye, S.L., Dinculescu, A., Hauswirth, W.W., Boye, S.E., Quantifying transduction efficiencies of unmodified and tyrosine capsid mutant AAV vectors in vitro using two ocular cell lines. Mol. Vis. 17 (2011), 1090–1102.
    • (2011) Mol. Vis. , vol.17 , pp. 1090-1102
    • Ryals, R.C.1    Boye, S.L.2    Dinculescu, A.3    Hauswirth, W.W.4    Boye, S.E.5
  • 33
    • 78649375117 scopus 로고    scopus 로고
    • High-efficiency transduction of fibroblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy
    • Li, M., Jayandharan, G.R., Li, B., Ling, C., Ma, W., Srivastava, A., Zhong, L., High-efficiency transduction of fibroblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy. Hum. Gene Ther. 21 (2010), 1527–1543.
    • (2010) Hum. Gene Ther. , vol.21 , pp. 1527-1543
    • Li, M.1    Jayandharan, G.R.2    Li, B.3    Ling, C.4    Ma, W.5    Srivastava, A.6    Zhong, L.7
  • 36
    • 0242406854 scopus 로고    scopus 로고
    • The brain's interstitial clefts and their glial walls
    • Brightman, M.W., The brain's interstitial clefts and their glial walls. J. Neurocytol. 31 (2002), 595–603.
    • (2002) J. Neurocytol. , vol.31 , pp. 595-603
    • Brightman, M.W.1
  • 37
    • 0038027345 scopus 로고    scopus 로고
    • RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism
    • Shi, W., Bartlett, J.S., RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism. Mol. Ther. 7 (2003), 515–525.
    • (2003) Mol. Ther. , vol.7 , pp. 515-525
    • Shi, W.1    Bartlett, J.S.2
  • 40
    • 4344566457 scopus 로고    scopus 로고
    • Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system
    • Burger, C., Gorbatyuk, O.S., Velardo, M.J., Peden, C.S., Williams, P., Zolotukhin, S., Reier, P.J., Mandel, R.J., Muzyczka, N., Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mol. Ther. 10 (2004), 302–317.
    • (2004) Mol. Ther. , vol.10 , pp. 302-317
    • Burger, C.1    Gorbatyuk, O.S.2    Velardo, M.J.3    Peden, C.S.4    Williams, P.5    Zolotukhin, S.6    Reier, P.J.7    Mandel, R.J.8    Muzyczka, N.9
  • 41
    • 64149106830 scopus 로고    scopus 로고
    • Comparison of transduction efficiency of recombinant AAV serotypes 1, 2, 5, and 8 in the rat nigrostriatal system
    • McFarland, N.R., Lee, J.S., Hyman, B.T., McLean, P.J., Comparison of transduction efficiency of recombinant AAV serotypes 1, 2, 5, and 8 in the rat nigrostriatal system. J. Neurochem. 109 (2009), 838–845.
    • (2009) J. Neurochem. , vol.109 , pp. 838-845
    • McFarland, N.R.1    Lee, J.S.2    Hyman, B.T.3    McLean, P.J.4
  • 43
    • 32944471565 scopus 로고    scopus 로고
    • Efficient neuronal gene transfer with AAV8 leads to neurotoxic levels of tau or green fluorescent proteins
    • Klein, R.L., Dayton, R.D., Leidenheimer, N.J., Jansen, K., Golde, T.E., Zweig, R.M., Efficient neuronal gene transfer with AAV8 leads to neurotoxic levels of tau or green fluorescent proteins. Mol. Ther. 13 (2006), 517–527.
    • (2006) Mol. Ther. , vol.13 , pp. 517-527
    • Klein, R.L.1    Dayton, R.D.2    Leidenheimer, N.J.3    Jansen, K.4    Golde, T.E.5    Zweig, R.M.6
  • 44
    • 84927911867 scopus 로고    scopus 로고
    • Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex
    • Watakabe, A., Ohtsuka, M., Kinoshita, M., Takaji, M., Isa, K., Mizukami, H., Ozawa, K., Isa, T., Yamamori, T., Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex. Neurosci. Res. 93 (2015), 144–157.
    • (2015) Neurosci. Res. , vol.93 , pp. 144-157
    • Watakabe, A.1    Ohtsuka, M.2    Kinoshita, M.3    Takaji, M.4    Isa, K.5    Mizukami, H.6    Ozawa, K.7    Isa, T.8    Yamamori, T.9
  • 45
    • 84925424833 scopus 로고    scopus 로고
    • A method for combining RNAscope in situ hybridization with immunohistochemistry in thick free-floating brain sections and primary neuronal cultures
    • Grabinski, T.M., Kneynsberg, A., Manfredsson, F.P., Kanaan, N.M., A method for combining RNAscope in situ hybridization with immunohistochemistry in thick free-floating brain sections and primary neuronal cultures. PLoS ONE, 10, 2015, e0120120.
    • (2015) PLoS ONE , vol.10 , pp. e0120120
    • Grabinski, T.M.1    Kneynsberg, A.2    Manfredsson, F.P.3    Kanaan, N.M.4
  • 50
    • 61649120100 scopus 로고    scopus 로고
    • Striatal readministration of rAAV vectors reveals an immune response against AAV2 capsids that can be circumvented
    • Peden, C.S., Manfredsson, F.P., Reimsnider, S.K., Poirier, A.E., Burger, C., Muzyczka, N., Mandel, R.J., Striatal readministration of rAAV vectors reveals an immune response against AAV2 capsids that can be circumvented. Mol. Ther. 17 (2009), 524–537.
    • (2009) Mol. Ther. , vol.17 , pp. 524-537
    • Peden, C.S.1    Manfredsson, F.P.2    Reimsnider, S.K.3    Poirier, A.E.4    Burger, C.5    Muzyczka, N.6    Mandel, R.J.7
  • 51
    • 84959142835 scopus 로고    scopus 로고
    • Continuous collection of adeno-associated virus from producer cell medium significantly increases total viral yield
    • Benskey, M.J., Sandoval, I.M., Manfredsson, F.P., Continuous collection of adeno-associated virus from producer cell medium significantly increases total viral yield. Hum. Gene Ther. Methods 27 (2016), 32–45.
    • (2016) Hum. Gene Ther. Methods , vol.27 , pp. 32-45
    • Benskey, M.J.1    Sandoval, I.M.2    Manfredsson, F.P.3
  • 53
    • 84948845949 scopus 로고    scopus 로고
    • Intraparenchymal stereotaxic delivery of rAAV and special considerations in vector handling
    • Benskey, M.J., Manfredsson, F.P., Intraparenchymal stereotaxic delivery of rAAV and special considerations in vector handling. Methods Mol. Biol. 1382 (2016), 199–215.
    • (2016) Methods Mol. Biol. , vol.1382 , pp. 199-215
    • Benskey, M.J.1    Manfredsson, F.P.2
  • 55
    • 84985280160 scopus 로고
    • The efficiency of systematic sampling in stereology and its prediction
    • Gundersen, H.J., Jensen, E.B., The efficiency of systematic sampling in stereology and its prediction. J. Microsc. 147 (1987), 229–263.
    • (1987) J. Microsc. , vol.147 , pp. 229-263
    • Gundersen, H.J.1    Jensen, E.B.2
  • 57
    • 34248172479 scopus 로고    scopus 로고
    • Interpretation of electron density with stereographic roadmap projections
    • Xiao, C., Rossmann, M.G., Interpretation of electron density with stereographic roadmap projections. J. Struct. Biol. 158 (2007), 182–187.
    • (2007) J. Struct. Biol. , vol.158 , pp. 182-187
    • Xiao, C.1    Rossmann, M.G.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.