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Volumn 19, Issue 2, 2012, Pages 176-181

Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9

Author keywords

Adeno associated virus; animal models of neurodegenerative disease; retina; systemic

Indexed keywords

PARVOVIRUS VECTOR; PHENYLALANINE; RHODOPSIN; TYROSINE;

EID: 84856975718     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2011.163     Document Type: Article
Times cited : (66)

References (26)
  • 1
    • 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, Roman AJ et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci USA 2008; 105: 15112-15117.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 15112-15117
    • Cideciyan, A.V.1    Aleman, T.S.2    Boye, S.L.3    Schwartz, S.B.4    Kaushal, S.5    Roman, A.J.6
  • 3
    • 50049136340 scopus 로고    scopus 로고
    • Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising
    • Bainbridge JW, Ali RR. Success in sight: the eyes have it! Ocular gene therapy trials for LCA look promising. Gene Therapy 2008; 15: 1191-1192.
    • (2008) Gene Therapy , vol.15 , pp. 1191-1192
    • Bainbridge, J.W.1    Ali, R.R.2
  • 4
    • 77952962177 scopus 로고    scopus 로고
    • Rod-derived cone viability factor for treating blinding diseases: From clinic to redox signaling
    • Léveillard T, Sahel J-A. Rod-derived cone viability factor for treating blinding diseases: from clinic to redox signaling. Science Trans Med 2010; 2: 26ps16.
    • (2010) Science Trans Med , vol.2
    • Léveillard, T.1    Sahel, J.-A.2
  • 5
    • 85049056931 scopus 로고    scopus 로고
    • Long-term retinal function and structure rescue using capsid mutant AAV8 Vector in the rd10 mouse, a model of recessive retinitis pigmentosa
    • Pang JJ, Dai X, Boye SE, Barone I, Boye SL, Mao S et al. Long-term retinal function and structure rescue using capsid mutant AAV8 Vector in the rd10 mouse, a model of recessive retinitis pigmentosa. Mol Ther 2009; 19: 232-242.
    • (2009) Mol Ther , vol.19 , pp. 232-242
    • Pang, J.J.1    Dai, X.2    Boye, S.E.3    Barone, I.4    Boye, S.L.5    Mao, S.6
  • 6
    • 0035664438 scopus 로고    scopus 로고
    • Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa
    • DOI 10.1006/mthe.2001.0498
    • Sanftner L. Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa. Mol Ther 2001; 4: 622-629. (Pubitemid 34026878)
    • (2001) Molecular Therapy , vol.4 , Issue.6 , pp. 622-629
    • Sanftner, L.H.M.1    Abel, H.2    Hauswirth, W.W.3    Flannery, J.G.4
  • 7
    • 73849132937 scopus 로고    scopus 로고
    • Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous
    • Dalkara D, Kolstad KD, Caporale N, Visel M, Klimczak RR, Schaffer DV et al. Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous. Mol Ther 2009; 17: 2096-2102.
    • (2009) Mol Ther , vol.17 , pp. 2096-2102
    • Dalkara, D.1    Kolstad, K.D.2    Caporale, N.3    Visel, M.4    Klimczak, R.R.5    Schaffer, D.V.6
  • 9
    • 79551634862 scopus 로고    scopus 로고
    • Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina
    • Petrs-Silva H, Dinculescu A, Li Q, Deng W-T, Pang J-j, Min S-H et al. Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina. Mol Ther 2009; 19: 293-301.
    • (2009) Mol Ther , vol.19 , pp. 293-301
    • Petrs-Silva, H.1    Dinculescu, A.2    Li, Q.3    Deng, W.-T.4    J-J, P.5    Min, S.-H.6
  • 10
    • 61649095399 scopus 로고    scopus 로고
    • High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors
    • Petrs-Silva H, Dinculescu A, Li Q, Min S-H, Chiodo V, Pang J-j et al. High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther 2009; 17: 463-471.
    • (2009) Mol Ther , vol.17 , pp. 463-471
    • Petrs-Silva, H.1    Dinculescu, A.2    Li, Q.3    Min, S.-H.4    Chiodo, V.5    J-J, P.6
  • 11
    • 70449338146 scopus 로고    scopus 로고
    • A novel adenoassociated viral variant for efficient and selective intravitreal transduction of rat Müller cells
    • Klimczak RR, Koerber JT, Dalkara D, Flannery JG, Schaffer DV. A novel adenoassociated viral variant for efficient and selective intravitreal transduction of rat Müller cells. PloS One 2009; 4: e7467.
    • (2009) PloS One , vol.4
    • Klimczak, R.R.1    Koerber, J.T.2    Dalkara, D.3    Flannery, J.G.4    Schaffer, D.V.5
  • 14
    • 0034870041 scopus 로고    scopus 로고
    • The fundamental plan of the retina
    • DOI 10.1038/nn0901-877
    • Masland RH. The fundamental plan of the retina. Nat Neurosci 2001; 4: 877-886. (Pubitemid 32801590)
    • (2001) Nature Neuroscience , vol.4 , Issue.9 , pp. 877-886
    • Masland, R.H.1
  • 15
    • 79959932534 scopus 로고    scopus 로고
    • Immune responses to AAV in clinical trials
    • Mingozzi F, High KA. Immune responses to AAV in clinical trials. Curr Gene Ther 2011; 11: 321-330.
    • (2011) Curr Gene Ther , vol.11 , pp. 321-330
    • Mingozzi, F.1    High, K.A.2
  • 16
    • 35848961457 scopus 로고    scopus 로고
    • Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration
    • DOI 10.1038/sj.gt.3303029, PII 3303029
    • Bostick B, Ghosh A, Yue Y, Long C, Duan D. Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration. Gene Therapy 2007; 14: 1605-1609. (Pubitemid 350056232)
    • (2007) Gene Therapy , vol.14 , Issue.22 , pp. 1605-1609
    • Bostick, B.1    Ghosh, A.2    Yue, Y.3    Long, C.4    Duan, D.5
  • 18
    • 77749249680 scopus 로고    scopus 로고
    • Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
    • Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM et al. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol 2010; 28: 271-274.
    • (2010) Nat Biotechnol , vol.28 , pp. 271-274
    • Foust, K.D.1    Wang, X.2    McGovern, V.L.3    Braun, L.4    Bevan, A.K.5    Haidet, A.M.6
  • 19
    • 79961032418 scopus 로고    scopus 로고
    • Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system
    • Zhang H, Yang B, Mu X, Ahmed SS, Su Q, He R et al. Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol Ther 2009; 19: 1440-1448.
    • (2009) Mol Ther , vol.19 , pp. 1440-1448
    • Zhang, H.1    Yang, B.2    Mu, X.3    Ahmed, S.S.4    Su, Q.5    He, R.6
  • 20
    • 80053923197 scopus 로고    scopus 로고
    • Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system
    • Rahim AA, Wong AM, Hoefer K, Buckley SM, Mattar CN, Cheng SH et al. Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system. FASEB J 2011; 25: 3505-3518.
    • (2011) FASEB J , vol.25 , pp. 3505-3518
    • Rahim, A.A.1    Wong, A.M.2    Hoefer, K.3    Buckley, S.M.4    Mattar, C.N.5    Cheng, S.H.6
  • 21
    • 33746874152 scopus 로고    scopus 로고
    • Adeno-associated Virus Serotypes: Vector Toolkit for Human Gene Therapy
    • DOI 10.1016/j.ymthe.2006.05.009, PII S1525001606002048
    • Wu Z, Asokan A, Samulski RJ. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 2006; 14: 316-327. (Pubitemid 44184972)
    • (2006) Molecular Therapy , vol.14 , Issue.3 , pp. 316-327
    • Wu, Z.1    Asokan, A.2    Samulski, R.J.3
  • 23
    • 16844379844 scopus 로고    scopus 로고
    • Viral vector mediated overexpression of human α-synuclein in the nigrostriatal dopaminergic neurons: A new model for Parkinson's disease
    • Maingay M, Romero-Ramos M. Viral vector mediated overexpression of human alphasynuclein in the nigrostriatal dopaminergic neurons: a new model for Parkinson's disease. CNS Spectr 2005; 10: 235-244. (Pubitemid 40489292)
    • (2005) CNS Spectrums , vol.10 , Issue.3 , pp. 235-244
    • Maingay, M.1    Romero-Ramos, M.2    Kirik, D.3
  • 24
    • 77957224859 scopus 로고    scopus 로고
    • Viral vector-mediated overexpression of alpha-synuclein as a progressive model of Parkinson's disease
    • Ulusoy A, Decressac M, Kirik D, Bjorklund A. Viral vector-mediated overexpression of alpha-synuclein as a progressive model of Parkinson's disease. Prog Brain Res 2010; 184: 89-111.
    • (2010) Prog Brain Res , vol.184 , pp. 89-111
    • Ulusoy, A.1    Decressac, M.2    Kirik, D.3    Bjorklund, A.4
  • 25
    • 67650218487 scopus 로고    scopus 로고
    • MicroRNAs-powerful repression comes from small RNAs
    • Ma C, Liu Y, He L. MicroRNAs-powerful repression comes from small RNAs. Science China C Life Sci 2009; 52: 323-330.
    • (2009) Science China C Life Sci , vol.52 , pp. 323-330
    • Ma, C.1    Liu, Y.2    He, L.3
  • 26
    • 34347252689 scopus 로고    scopus 로고
    • Production and characterization of adeno-associated viral vectors
    • Grieger JC, Choi VW, Samulski RJ. Production and characterization of adeno-associated viral vectors. Nat Protoc 2006; 1: 1412-1428.
    • (2006) Nat Protoc , vol.1 , pp. 1412-1428
    • Grieger, J.C.1    Choi, V.W.2    Samulski, R.J.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.