메뉴 건너뛰기




Volumn 6, Issue 7, 2011, Pages

MicroRNA-Restricted transgene expression in the retina

Author keywords

[No Author keywords available]

Indexed keywords

MICRORNA; MICRORNA 124; MICRORNA 204; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG; ENHANCED GREEN FLUORESCENT PROTEIN; GREEN FLUORESCENT PROTEIN;

EID: 79960788931     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0022166     Document Type: Article
Times cited : (55)

References (52)
  • 1
    • 78751477191 scopus 로고    scopus 로고
    • Gene silencing by microRNAs: contributions of translational repression and mRNA decay
    • Huntzinger E, Izaurralde E, (2011) Gene silencing by microRNAs: contributions of translational repression and mRNA decay. Nat Rev Genet 12: 99-110.
    • (2011) Nat Rev Genet , vol.12 , pp. 99-110
    • Huntzinger, E.1    Izaurralde, E.2
  • 2
    • 34250877841 scopus 로고    scopus 로고
    • A mammalian microRNA expression atlas based on small RNA library sequencing
    • Landgraf P, Rusu M, Sheridan R, Sewer A, Iovino N, et al. (2007) A mammalian microRNA expression atlas based on small RNA library sequencing. Cell 129: 1401-1414.
    • (2007) Cell , vol.129 , pp. 1401-1414
    • Landgraf, P.1    Rusu, M.2    Sheridan, R.3    Sewer, A.4    Iovino, N.5
  • 3
    • 67651087321 scopus 로고    scopus 로고
    • Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications
    • Brown BD, Naldini L, (2009) Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nat Rev Genet 10: 578-585.
    • (2009) Nat Rev Genet , vol.10 , pp. 578-585
    • Brown, B.D.1    Naldini, L.2
  • 4
    • 36849060584 scopus 로고    scopus 로고
    • Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
    • Brown BD, Gentner B, Cantore A, Colleoni S, Amendola M, et al. (2007) Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol 25: 1457-1467.
    • (2007) Nat Biotechnol , vol.25 , pp. 1457-1467
    • Brown, B.D.1    Gentner, B.2    Cantore, A.3    Colleoni, S.4    Amendola, M.5
  • 5
    • 64749092065 scopus 로고    scopus 로고
    • Engineered lentiviral vector targeting astrocytes in vivo
    • Colin A, Faideau M, Dufour N, Auregan G, Hassig R, et al. (2009) Engineered lentiviral vector targeting astrocytes in vivo. Glia 57: 667-679.
    • (2009) Glia , vol.57 , pp. 667-679
    • Colin, A.1    Faideau, M.2    Dufour, N.3    Auregan, G.4    Hassig, R.5
  • 6
    • 61749100637 scopus 로고    scopus 로고
    • Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras
    • Papapetrou EP, Kovalovsky D, Beloeil L, Sant'angelo D, Sadelain M, (2009) Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras. J Clin Invest 119: 157-168.
    • (2009) J Clin Invest , vol.119 , pp. 157-168
    • Papapetrou, E.P.1    Kovalovsky, D.2    Beloeil, L.3    Sant'angelo, D.4    Sadelain, M.5
  • 7
    • 77954923288 scopus 로고    scopus 로고
    • Tracking differentiating neural progenitors in pluripotent cultures using microRNA-regulated lentiviral vectors
    • Sachdeva R, Jonsson ME, Nelander J, Kirkeby A, Guibentif C, et al. (2010) Tracking differentiating neural progenitors in pluripotent cultures using microRNA-regulated lentiviral vectors. Proc Natl Acad Sci U S A 107: 11602-11607.
    • (2010) Proc Natl Acad Sci U S A , vol.107 , pp. 11602-11607
    • Sachdeva, R.1    Jonsson, M.E.2    Nelander, J.3    Kirkeby, A.4    Guibentif, C.5
  • 8
    • 79952193362 scopus 로고    scopus 로고
    • MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression
    • Xie J, Xie Q, Zhang H, Ameres SL, Hung JH, et al. (2011) MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression. Mol Ther 19: 526-535.
    • (2011) Mol Ther , vol.19 , pp. 526-535
    • Xie, J.1    Xie, Q.2    Zhang, H.3    Ameres, S.L.4    Hung, J.H.5
  • 9
    • 36849053006 scopus 로고    scopus 로고
    • A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
    • Brown BD, Cantore A, Annoni A, Sergi LS, Lombardo A, et al. (2007) A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood 110: 4144-4152.
    • (2007) Blood , vol.110 , pp. 4144-4152
    • Brown, B.D.1    Cantore, A.2    Annoni, A.3    Sergi, L.S.4    Lombardo, A.5
  • 10
    • 78449270317 scopus 로고    scopus 로고
    • Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy
    • Gentner B, Visigalli I, Hiramatsu H, Lechman E, Ungari S, et al. (2010) Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med 2: 58ra84.
    • (2010) Sci Transl Med , vol.2
    • Gentner, B.1    Visigalli, I.2    Hiramatsu, H.3    Lechman, E.4    Ungari, S.5
  • 13
    • 58149484922 scopus 로고    scopus 로고
    • Ocular gene therapy: current progress and future prospects
    • Colella P, Cotugno G, Auricchio A, (2009) Ocular gene therapy: current progress and future prospects. Trends Mol Med 15: 23-31.
    • (2009) Trends Mol Med , vol.15 , pp. 23-31
    • Colella, P.1    Cotugno, G.2    Auricchio, A.3
  • 14
    • 44249120315 scopus 로고    scopus 로고
    • Effect of gene therapy on visual function in Leber's congenital amaurosis
    • Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, et al. (2008) Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 358: 2231-2239.
    • (2008) N Engl J Med , vol.358 , pp. 2231-2239
    • Bainbridge, J.W.1    Smith, A.J.2    Barker, S.S.3    Robbie, S.4    Henderson, R.5
  • 15
    • 54449085219 scopus 로고    scopus 로고
    • Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
    • Cideciyan AV, Aleman TS, Boye SL, Schwartz SB, Kaushal S, et al. (2008) Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A 105: 15112-15117.
    • (2008) Proc Natl Acad Sci U S A , vol.105 , pp. 15112-15117
    • Cideciyan, A.V.1    Aleman, T.S.2    Boye, S.L.3    Schwartz, S.B.4    Kaushal, S.5
  • 16
    • 70349105559 scopus 로고    scopus 로고
    • Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year
    • Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, et al. (2009) Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum Gene Ther 20: 999-1004.
    • (2009) Hum Gene Ther , vol.20 , pp. 999-1004
    • Cideciyan, A.V.1    Hauswirth, W.W.2    Aleman, T.S.3    Kaushal, S.4    Schwartz, S.B.5
  • 18
    • 54949104686 scopus 로고    scopus 로고
    • Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial
    • Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, et al. (2008) Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther 19: 979-990.
    • (2008) Hum Gene Ther , vol.19 , pp. 979-990
    • Hauswirth, W.W.1    Aleman, T.S.2    Kaushal, S.3    Cideciyan, A.V.4    Schwartz, S.B.5
  • 19
    • 70350620424 scopus 로고    scopus 로고
    • Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
    • Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, et al. (2009) Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 374: 1597-1605.
    • (2009) Lancet , vol.374 , pp. 1597-1605
    • Maguire, A.M.1    High, K.A.2    Auricchio, A.3    Wright, J.F.4    Pierce, E.A.5
  • 21
    • 77649242176 scopus 로고    scopus 로고
    • Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration
    • Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, et al. (2010) Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther 18: 643-650.
    • (2010) Mol Ther , vol.18 , pp. 643-650
    • Simonelli, F.1    Maguire, A.M.2    Testa, F.3    Pierce, E.A.4    Mingozzi, F.5
  • 22
    • 38749087929 scopus 로고    scopus 로고
    • Versatility of AAV vectors for retinal gene transfer
    • Surace EM, Auricchio A, (2008) Versatility of AAV vectors for retinal gene transfer. Vision Res 48: 353-359.
    • (2008) Vision Res , vol.48 , pp. 353-359
    • Surace, E.M.1    Auricchio, A.2
  • 23
    • 0034613191 scopus 로고    scopus 로고
    • The upstream region of the Rpe65 gene confers retinal pigment epithelium-specific expression in vivo and in vitro and contains critical octamer and E-box binding sites
    • Boulanger A, Liu S, Henningsgaard AA, Yu S, Redmond TM, (2000) The upstream region of the Rpe65 gene confers retinal pigment epithelium-specific expression in vivo and in vitro and contains critical octamer and E-box binding sites. J Biol Chem 275: 31274-31282.
    • (2000) J Biol Chem , vol.275 , pp. 31274-31282
    • Boulanger, A.1    Liu, S.2    Henningsgaard, A.A.3    Yu, S.4    Redmond, T.M.5
  • 24
  • 26
    • 33847746661 scopus 로고    scopus 로고
    • Identification and characterization of microRNAs expressed in the mouse eye
    • Karali M, Peluso I, Marigo V, Banfi S, (2007) Identification and characterization of microRNAs expressed in the mouse eye. Invest Ophthalmol Vis Sci 48: 509-515.
    • (2007) Invest Ophthalmol Vis Sci , vol.48 , pp. 509-515
    • Karali, M.1    Peluso, I.2    Marigo, V.3    Banfi, S.4
  • 27
    • 35148862553 scopus 로고    scopus 로고
    • Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors
    • Allocca M, Mussolino C, Garcia-Hoyos M, Sanges D, Iodice C, et al. (2007) Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. J Virol 81: 11372-11380.
    • (2007) J Virol , vol.81 , pp. 11372-11380
    • Allocca, M.1    Mussolino, C.2    Garcia-Hoyos, M.3    Sanges, D.4    Iodice, C.5
  • 28
    • 0035894739 scopus 로고    scopus 로고
    • Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model
    • Auricchio A, Kobinger G, Anand V, Hildinger M, O'Connor E, et al. (2001) Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum Mol Genet 10: 3075-3081.
    • (2001) Hum Mol Genet , vol.10 , pp. 3075-3081
    • Auricchio, A.1    Kobinger, G.2    Anand, V.3    Hildinger, M.4    O'Connor, E.5
  • 29
    • 0142090629 scopus 로고    scopus 로고
    • Adeno-associated viral vectors for retinal gene transfer
    • Surace EM, Auricchio A, (2003) Adeno-associated viral vectors for retinal gene transfer. Prog Retin Eye Res 22: 705-719.
    • (2003) Prog Retin Eye Res , vol.22 , pp. 705-719
    • Surace, E.M.1    Auricchio, A.2
  • 31
    • 33748310866 scopus 로고    scopus 로고
    • Detection of mammalian microRNA expression by in situ hybridization with RNA oligonucleotides
    • Deo M, Yu JY, Chung KH, Tippens M, Turner DL, (2006) Detection of mammalian microRNA expression by in situ hybridization with RNA oligonucleotides. Dev Dyn 235: 2538-2548.
    • (2006) Dev Dyn , vol.235 , pp. 2538-2548
    • Deo, M.1    Yu, J.Y.2    Chung, K.H.3    Tippens, M.4    Turner, D.L.5
  • 37
    • 0036310829 scopus 로고    scopus 로고
    • Distribution and density of medium- and short-wavelength selective cones in the domestic pig retina
    • Hendrickson A, Hicks D, (2002) Distribution and density of medium- and short-wavelength selective cones in the domestic pig retina. Exp Eye Res 74: 435-444.
    • (2002) Exp Eye Res , vol.74 , pp. 435-444
    • Hendrickson, A.1    Hicks, D.2
  • 40
    • 35148819156 scopus 로고    scopus 로고
    • Prediction and verification of miRNA expression in human and rat retinas
    • Arora A, McKay GJ, Simpson DA, (2007) Prediction and verification of miRNA expression in human and rat retinas. Invest Ophthalmol Vis Sci 48: 3962-3967.
    • (2007) Invest Ophthalmol Vis Sci , vol.48 , pp. 3962-3967
    • Arora, A.1    McKay, G.J.2    Simpson, D.A.3
  • 41
    • 0033556147 scopus 로고    scopus 로고
    • Identification of a cellubrevin/vesicle associated membrane protein 3 homologue in human platelets
    • Bernstein AM, Whiteheart SW, (1999) Identification of a cellubrevin/vesicle associated membrane protein 3 homologue in human platelets. Blood 93: 571-579.
    • (1999) Blood , vol.93 , pp. 571-579
    • Bernstein, A.M.1    Whiteheart, S.W.2
  • 42
    • 43749093884 scopus 로고    scopus 로고
    • Dynamic expression of the retinoic acid-synthesizing enzyme retinol dehydrogenase 10 (rdh10) in the developing mouse brain and sensory organs
    • Romand R, Kondo T, Cammas L, Hashino E, Dolle P, (2008) Dynamic expression of the retinoic acid-synthesizing enzyme retinol dehydrogenase 10 (rdh10) in the developing mouse brain and sensory organs. J Comp Neurol 508: 879-892.
    • (2008) J Comp Neurol , vol.508 , pp. 879-892
    • Romand, R.1    Kondo, T.2    Cammas, L.3    Hashino, E.4    Dolle, P.5
  • 44
    • 33645720940 scopus 로고    scopus 로고
    • Does recombinant adeno-associated virus-vectored proximal region of mouse rhodopsin promoter support only rod-type specific expression in vivo?
    • Glushakova LG, Timmers AM, Issa TM, Cortez NG, Pang J, et al. (2006) Does recombinant adeno-associated virus-vectored proximal region of mouse rhodopsin promoter support only rod-type specific expression in vivo? Mol Vis 12: 298-309.
    • (2006) Mol Vis , vol.12 , pp. 298-309
    • Glushakova, L.G.1    Timmers, A.M.2    Issa, T.M.3    Cortez, N.G.4    Pang, J.5
  • 45
    • 0026522293 scopus 로고
    • Rhodopsin and phototransduction: a model system for G protein-linked receptors
    • Hargrave PA, McDowell JH, (1992) Rhodopsin and phototransduction: a model system for G protein-linked receptors. Faseb J 6: 2323-2331.
    • (1992) Faseb J , vol.6 , pp. 2323-2331
    • Hargrave, P.A.1    McDowell, J.H.2
  • 46
    • 3242686879 scopus 로고    scopus 로고
    • The microphthalmia transcription factor (Mitf) controls expression of the ocular albinism type 1 gene: link between melanin synthesis and melanosome biogenesis
    • Vetrini F, Auricchio A, Du J, Angeletti B, Fisher DE, et al. (2004) The microphthalmia transcription factor (Mitf) controls expression of the ocular albinism type 1 gene: link between melanin synthesis and melanosome biogenesis. Mol Cell Biol 24: 6550-6559.
    • (2004) Mol Cell Biol , vol.24 , pp. 6550-6559
    • Vetrini, F.1    Auricchio, A.2    du, J.3    Angeletti, B.4    Fisher, D.E.5
  • 47
    • 28444442243 scopus 로고    scopus 로고
    • Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness
    • Acland GM, Aguirre GD, Bennett J, Aleman TS, Cideciyan AV, et al. (2005) Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol Ther 12: 1072-1082.
    • (2005) Mol Ther , vol.12 , pp. 1072-1082
    • Acland, G.M.1    Aguirre, G.D.2    Bennett, J.3    Aleman, T.S.4    Cideciyan, A.V.5
  • 48
    • 39849110435 scopus 로고    scopus 로고
    • Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer
    • Bennicelli J, Wright JF, Komaromy A, Jacobs JB, Hauck B, et al. (2008) Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol Ther 16: 458-465.
    • (2008) Mol Ther , vol.16 , pp. 458-465
    • Bennicelli, J.1    Wright, J.F.2    Komaromy, A.3    Jacobs, J.B.4    Hauck, B.5
  • 51
    • 0036235037 scopus 로고    scopus 로고
    • Retinoic acid upregulates cone arrestin expression in retinoblastoma cells through a Cis element in the distal promoter region
    • Li A, Zhu X, Craft CM, (2002) Retinoic acid upregulates cone arrestin expression in retinoblastoma cells through a Cis element in the distal promoter region. Invest Ophthalmol Vis Sci 43: 1375-1383.
    • (2002) Invest Ophthalmol Vis Sci , vol.43 , pp. 1375-1383
    • Li, A.1    Zhu, X.2    Craft, C.M.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.