메뉴 건너뛰기




Volumn 23, Issue 8, 2012, Pages 847-858

Efficacy of codelivery of dual AAV2/5 vectors in the murine retina and hippocampus

Author keywords

[No Author keywords available]

Indexed keywords

GREEN FLUORESCENT PROTEIN; MESSENGER RNA; RED FLUORESCENT PROTEIN; VIRUS VECTOR;

EID: 84865219047     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2011.142     Document Type: Article
Times cited : (31)

References (62)
  • 1
    • 43049119982 scopus 로고    scopus 로고
    • Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice
    • Allocca, M., Doria, M., Petrillo, M., et al. (2008). Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J. Clin. Invest. 118, 1955-1964.
    • (2008) J. Clin. Invest. , vol.118 , pp. 1955-1964
    • Allocca, M.1    Doria, M.2    Petrillo, M.3
  • 2
    • 0035894739 scopus 로고    scopus 로고
    • Exchange of surface proteins imparts on viral cellular specificity and transduction characteristics: The retina as a model
    • Auricchio, A., Kobinger, G., Anand, V., et al. (2001). Exchange of surface proteins imparts on viral 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
  • 3
    • 44249120315 scopus 로고    scopus 로고
    • Effect of gene therapy on visual function in Leber's congenital amau-rosis
    • Bainbridge, J.W., Smith, A.J., Barker, S.S., et al. (2008). Effect of gene therapy on visual function in Leber's congenital amau-rosis. N. Engl. J. Med. 22;358, 2231-2239.
    • (2008) N. Engl. J. Med. , vol.22 , Issue.358 , pp. 2231-2239
    • Bainbridge, J.W.1    Smith, A.J.2    Barker, S.S.3
  • 4
    • 39849110435 scopus 로고    scopus 로고
    • Reversal of blindness in animal models of Leber congenital amaurosis using optimized AAV2-mediatedgenetransfer
    • Bennicelli, J., Wright, J.F., Komaromy, A., et al. (2008). Reversal of blindness in animal models of Leber congenital amaurosis using optimized AAV2-mediatedgenetransfer. Mol. Ther.16, 458-465.
    • (2008) Mol. Ther. , vol.16 , pp. 458-465
    • Bennicelli, J.1    Wright, J.F.2    Komaromy, A.3
  • 5
    • 33749236815 scopus 로고    scopus 로고
    • In contrast to AAV-mediated Cntf expression, AAV-mediated Gdnf expression enhances gene replacement therapy in rodent models of retinal degeneration
    • Buch, P.K., MacLaren, R.E., Durán, Y., et al. (2006). In contrast to AAV-mediated Cntf expression, AAV-mediated Gdnf expression enhances gene replacement therapy in rodent models of retinal degeneration. Mol. Ther. 14, 700-709.
    • (2006) Mol. Ther. , vol.14 , pp. 700-709
    • Buch, P.K.1    MacLaren, R.E.2    Durán, Y.3
  • 6
    • 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., et al. (2004). 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, 302-317.
    • (2004) Mol. Ther. , vol.10 , pp. 302-317
    • Burger, C.1    Gorbatyuk, O.S.2    Velardo, M.J.3
  • 7
    • 77954847711 scopus 로고    scopus 로고
    • Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa
    • Busskamp, V., Duebel, J., Balya, D., et al. (2010). Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa. Science 329, 413-417.
    • (2010) Science , vol.329 , pp. 413-417
    • Busskamp, V.1    Duebel, J.2    Balya, D.3
  • 8
    • 79959924108 scopus 로고    scopus 로고
    • LiGluR restores visual responses in rodent models of inherited blindness
    • Caporale, N., Kolstad, K.D., Lee, T., et al. (2011). LiGluR restores visual responses in rodent models of inherited blindness. Mol. Ther. 19, 1212-1219.
    • (2011) Mol. Ther. , vol.19 , pp. 1212-1219
    • Caporale, N.1    Kolstad, K.D.2    Lee, T.3
  • 9
    • 77954847056 scopus 로고    scopus 로고
    • Neuroscience: Seeing the light of day
    • Cepko, C. (2010). Neuroscience: Seeing the light of day. Science 329, 403-404.
    • (2010) Science , vol.329 , pp. 403-404
    • Cepko, C.1
  • 10
    • 63949087144 scopus 로고    scopus 로고
    • Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy
    • Chadderton, N., Millington-Ward, S., Palfi, A., et al. (2009). Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy. Mol. Ther. 17, 593-599.
    • (2009) Mol. Ther. , vol.17 , pp. 593-599
    • Chadderton, N.1    Millington-Ward, S.2    Palfi, A.3
  • 11
    • 73449128979 scopus 로고    scopus 로고
    • Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
    • Christine, C.W., Starr, P.A., Larson, P.S., et al. (2009). Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology 73, 1662-1669.
    • (2009) Neurology , vol.73 , pp. 1662-1669
    • Christine, C.W.1    Starr, P.A.2    Larson, P.S.3
  • 12
    • 74049092844 scopus 로고    scopus 로고
    • Characterization of genome integrity for oversized recombinant AAV vector
    • Dong, B., Nakai, H., and Xiao, W. (2010). Characterization of genome integrity for oversized recombinant AAV vector. Mol. Ther. 18, 87-92.
    • (2010) Mol. Ther. , vol.18 , pp. 87-92
    • Dong, B.1    Nakai, H.2    Xiao, W.3
  • 13
    • 0029849257 scopus 로고    scopus 로고
    • Quantitative analysis of the packaging capacity of recombinant adeno-associated virus
    • Dong, J.Y., Fan, P.D., and Frizzell, R.A. (1996). Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum. Gene Ther. 7, 2101-2112.
    • (1996) Hum. Gene Ther. , vol.7 , pp. 2101-2112
    • Dong, J.Y.1    Fan, P.D.2    Frizzell, R.A.3
  • 14
    • 32944467645 scopus 로고    scopus 로고
    • Activation of multiple pathways during photoreceptor apoptosis in the rd mouse
    • Doonan, F., Donovan, M., and Cotter, T.G. (2005). Activation of multiple pathways during photoreceptor apoptosis in the rd mouse. Invest. Ophthalmol. Vis. Sci. 46, 3530-3538.
    • (2005) Invest. Ophthalmol. Vis. Sci. , vol.46 , pp. 3530-3538
    • Doonan, F.1    Donovan, M.2    Cotter, T.G.3
  • 15
    • 0034121397 scopus 로고    scopus 로고
    • A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermo-lecular cis activation
    • Duan, D., Yue, Y., Yan, Z., and Engelhardt, J.F. (2000). A new dual-vector approach to enhance recombinant adeno-associated virus-mediated gene expression through intermo-lecular cis activation. Nat. Med. 6, 595-598.
    • (2000) Nat. Med. , vol.6 , pp. 595-598
    • Duan, D.1    Yue, Y.2    Yan, Z.3    Engelhardt, J.F.4
  • 16
    • 0032553713 scopus 로고    scopus 로고
    • Behavioral recovery in 6-hydroxydopamine-lesioned rats by cotransduction of striatum with tyrosine hydroxylase and aromatic l-amino acid decarboxylase genes using two separate adeno-associated virus vectors
    • Fan, D.S., Ogawa, M., Fujimoto, K.I., et al. (1998). Behavioral recovery in 6-hydroxydopamine-lesioned rats by cotransduction of striatum with tyrosine hydroxylase and aromatic l-amino acid decarboxylase genes using two separate adeno-associated virus vectors. Hum. Gene Ther. 9, 2527-2535.
    • (1998) Hum. Gene Ther. , vol.9 , pp. 2527-2535
    • Fan, D.S.1    Ogawa, M.2    Fujimoto, K.I.3
  • 17
    • 78651312593 scopus 로고    scopus 로고
    • Gene therapeutic approaches for dominant retinopathies
    • Farrar, G.J., Palfi, A., and O'Reilly, M. (2010). Gene therapeutic approaches for dominant retinopathies. Curr. Gene Ther. 10, 381-388.
    • (2010) Curr. Gene Ther. , vol.10 , pp. 381-388
    • Farrar, G.J.1    Palfi, A.2    O'Reilly, M.3
  • 19
    • 33645223689 scopus 로고    scopus 로고
    • Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle
    • Ghosh, A., Yue, Y., and Duan, D. (2006). Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle. J. Gene Med. 8, 298-305.
    • (2006) J. Gene Med. , vol.8 , pp. 298-305
    • Ghosh, A.1    Yue, Y.2    Duan, D.3
  • 20
    • 70449119011 scopus 로고    scopus 로고
    • Systemic trans-splicing adeno-associated viral delivery efficiently transduces the heart of adult mdx mouse, a model for Du-chenne muscular dystrophy
    • Ghosh, A., Yue, Y., Shin, J.H., and Duan, D. (2009). Systemic trans-splicing adeno-associated viral delivery efficiently transduces the heart of adult mdx mouse, a model for Du-chenne muscular dystrophy. Hum. Gene Ther. 20, 1319-1328.
    • (2009) Hum. Gene Ther. , vol.20 , pp. 1319-1328
    • Ghosh, A.1    Yue, Y.2    Shin, J.H.3    Duan, D.4
  • 21
    • 0035989639 scopus 로고    scopus 로고
    • Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene
    • Halbert, C.L., Allen, J.M., and Miller, A.D. (2002). Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat. Biotechnol. 20, 697-701.
    • (2002) Nat. Biotechnol. , vol.20 , pp. 697-701
    • Halbert, C.L.1    Allen, J.M.2    Miller, A.D.3
  • 24
    • 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, W.W., Aleman, T.S., Kaushal, S., 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
  • 25
    • 33744495152 scopus 로고    scopus 로고
    • Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
    • Jacobson, S.G., Acland, G.M., Aguirre, G.D., et al. (2006). Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Mol. Ther. 13, 1074-1084.
    • (2006) Mol. Ther. , vol.13 , pp. 1074-1084
    • Jacobson, S.G.1    Acland, G.M.2    Aguirre, G.D.3
  • 26
    • 0032212151 scopus 로고    scopus 로고
    • The major cell populations of the mouse retina
    • Jeon, C.J., Strettoi, E., and Masland, R.H. (1998). The major cell populations of the mouse retina. J. Neurosci. 18, 8936-8946.
    • (1998) J. Neurosci. , vol.18 , pp. 8936-8946
    • Jeon, C.J.1    Strettoi, E.2    Masland, R.H.3
  • 27
    • 79954650563 scopus 로고    scopus 로고
    • State-of - The-art gene-based therapies: The road ahead
    • Kay, M.A. (2011). State-of-the-art gene-based therapies: The road ahead. Nat. Rev. Genet. 12, 316-328.
    • (2011) Nat. Rev. Genet. , vol.12 , pp. 316-328
    • Kay, M.A.1
  • 28
    • 24144492099 scopus 로고    scopus 로고
    • Coupling of RNAi-mediated target downregulation with gene replacement
    • Kim, D.H., and Rossi, J.J. (2003). Coupling of RNAi-mediated target downregulation with gene replacement. Antisense Nucleic Acid Drug Dev. 13, 151-155.
    • (2003) Antisense Nucleic Acid Drug Dev. , vol.13 , pp. 151-155
    • Kim, D.H.1    Rossi, J.J.2
  • 29
    • 35248831880 scopus 로고    scopus 로고
    • An-tioxidants slow photoreceptor cell death in mouse models of retinitis pigmentosa
    • Komeima, K., Rogers, B.S., and Campochiaro, P.A. (2007). An-tioxidants slow photoreceptor cell death in mouse models of retinitis pigmentosa. J. Cell Physiol. 213, 809-815.
    • (2007) J. Cell Physiol. , vol.213 , pp. 809-815
    • Komeima, K.1    Rogers, B.S.2    Campochiaro, P.A.3
  • 30
    • 79951878087 scopus 로고    scopus 로고
    • In vivo application of an RNAi strategy for the selective suppression of a mutant allele
    • Kubodera, T., Yamada, H., Anzai, M., et al. (2011). In vivo application of an RNAi strategy for the selective suppression of a mutant allele. Hum. Gene Ther. 22, 27-34.
    • (2011) Hum. Gene Ther. , vol.22 , pp. 27-34
    • Kubodera, T.1    Yamada, H.2    Anzai, M.3
  • 31
    • 44349103098 scopus 로고    scopus 로고
    • Light-activated channels targeted to on bipolar cells restore visual function in retinal degeneration
    • Lagali, P.S., Balya, D., Awatramani, G.B., et al. (2008). Light-activated channels targeted to ON bipolar cells restore visual function in retinal degeneration. Nat. Neurosci. 11, 667-675.
    • (2008) Nat. Neurosci. , vol.11 , pp. 667-675
    • Lagali, P.S.1    Balya, D.2    Awatramani, G.B.3
  • 32
    • 74149093851 scopus 로고    scopus 로고
    • Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome ‡ 8.2 kb
    • Lai, Y., Yue, Y., and Duan, D. (2010). Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome ‡ 8.2 kb. Mol. Ther. 18, 75-79.
    • (2010) Mol. Ther. , vol.18 , pp. 75-79
    • Lai, Y.1    Yue, Y.2    Duan, D.3
  • 33
    • 45749099260 scopus 로고    scopus 로고
    • Engineering and selection of shuffled AAV genomes: A new strategy for producing targeted biological nanoparticles
    • Li, W., Asokan, A., Wu, Z., et al. (2008). Engineering and selection of shuffled AAV genomes: A new strategy for producing targeted biological nanoparticles. Mol. Ther. 16, 1252-1260.
    • (2008) Mol. Ther. , vol.16 , pp. 1252-1260
    • Li, W.1    Asokan, A.2    Wu, Z.3
  • 34
    • 28844437489 scopus 로고    scopus 로고
    • Viral-mediated temporally controlled dopamine production in a rat model of Parkinson disease
    • Li, X.G., Okada, T., Kodera, M., et al. (2006). Viral-mediated temporally controlled dopamine production in a rat model of Parkinson disease. Mol. Ther. 13, 160-166.
    • (2006) Mol. Ther. , vol.13 , pp. 160-166
    • Li, X.G.1    Okada, T.2    Kodera, M.3
  • 35
    • 77949751399 scopus 로고    scopus 로고
    • CNTF induces regeneration of cone outer segments in a rat model of retinal degeneration
    • Li, Y., Tao, W., Luo, L., et al. (2010). CNTF induces regeneration of cone outer segments in a rat model of retinal degeneration. PLoS One 5, e9495.
    • (2010) PLoS One , vol.5
    • Li, Y.1    Tao, W.2    Luo, L.3
  • 36
    • 79551622680 scopus 로고    scopus 로고
    • Pre-clinical safety evaluation of AAV2-sFLT01: A gene therapy for age-related macular degeneration
    • MacLachlan, T.K., Lukason, M., Collins, M., et al. (2011). Pre-clinical safety evaluation of AAV2-sFLT01: A gene therapy for age-related macular degeneration. Mol. Ther. 19, 326-334.
    • (2011) Mol. Ther. , vol.19 , pp. 326-334
    • MacLachlan, T.K.1    Lukason, M.2    Collins, M.3
  • 37
    • 44249085878 scopus 로고    scopus 로고
    • Safety and efficacy of gene transfer for Leber's congenital amaurosis
    • Maguire, A.M., Simonelli, F., Pierce, E.A., et al. (2008). Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med. 358, 2240-2248.
    • (2008) N. Engl. J. Med. , vol.358 , pp. 2240-2248
    • Maguire, A.M.1    Simonelli, F.2    Pierce, E.A.3
  • 38
    • 32344438754 scopus 로고    scopus 로고
    • Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
    • Maheshri, N., Koerber, J.T., Kaspar, B.K., and Schaffer, D.V. (2006). Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat. Biotechnol. 24, 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
  • 39
    • 77950838482 scopus 로고    scopus 로고
    • CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease
    • Mandel, R.J. (2010). CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease. Curr. Opin. Mol. Ther. 12, 240-247.
    • (2010) Curr. Opin. Mol. Ther. , vol.12 , pp. 240-247
    • Mandel, R.J.1
  • 40
    • 84860175219 scopus 로고    scopus 로고
    • Long term rescue of retinal structure and function in P23H RHO trans-genic mice by rhodopsin RNA replacement with a single AAV vector
    • Mao, H., Gorbatyuk, M.S., Rossmiller, B., et al. (2012). Long term rescue of retinal structure and function in P23H RHO trans-genic mice by rhodopsin RNA replacement with a single AAV vector. Hum. Gene Ther. 23, 356-366.
    • (2012) Hum. Gene Ther. , vol.23 , pp. 356-366
    • Mao, H.1    Gorbatyuk, M.S.2    Rossmiller, B.3
  • 41
    • 41949110690 scopus 로고    scopus 로고
    • Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: An open-label, phase i trial
    • Marks, W.J., Jr., Ostrem, J.L., Verhagen, L., et al. (2008). Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: An open-label, phase I trial. Lancet Neurol. 7, 400-408.
    • (2008) Lancet Neurol. , vol.7 , pp. 400-408
    • Marks, Jr.W.J.1    Ostrem, J.L.2    Verhagen, L.3
  • 42
    • 54849162100 scopus 로고    scopus 로고
    • Self-complementary AAV vectors: Advances and applications
    • McCarty, D.M. (2008). Self-complementary AAV vectors: Advances and applications. Mol. Ther. 16, 1648-1656.
    • (2008) Mol. Ther. , vol.16 , pp. 1648-1656
    • McCarty, D.M.1
  • 43
    • 75149181068 scopus 로고    scopus 로고
    • AAV-mediated chronic over-expression of SNAP-25 in adult rat dorsal hippocampus impairs memory-associated synaptic plasticity
    • McKee, A.G., Loscher, J.S., O'Sullivan, N.C., et al. (2010). AAV-mediated chronic over-expression of SNAP-25 in adult rat dorsal hippocampus impairs memory-associated synaptic plasticity. J. Neurochem. 112, 991-1004
    • (2010) J. Neurochem. , vol.112 , pp. 991-1004
    • McKee, A.G.1    Loscher, J.S.2    O'Sullivan, N.C.3
  • 44
    • 9844225036 scopus 로고    scopus 로고
    • Stratagems in vitro for gene therapies directed to dominant mutations
    • Millington-Ward, S., O'Neill, B., Tuohy, G., et al. (1997). Stratagems in vitro for gene therapies directed to dominant mutations. Hum. Mol. Genet. 6, 1415-1426.
    • (1997) Hum. Mol. Genet. , vol.6 , pp. 1415-1426
    • Millington-Ward, S.1    O'Neill, B.2    Tuohy, G.3
  • 45
    • 79953314650 scopus 로고    scopus 로고
    • Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa
    • Millington-Ward, S., Chadderton, N., O'Reilly, M., et al. (2011). Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa. Mol. Ther. 19, 642-649.
    • (2011) Mol. Ther. , vol.19 , pp. 642-649
    • Millington-Ward, S.1    Chadderton, N.2    O'Reilly, M.3
  • 46
    • 78049478882 scopus 로고    scopus 로고
    • Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: A strategy for broad clinical application
    • Monahan, P.E., Lothrop, C.D., Sun, J., et al. (2010). Proteasome inhibitors enhance gene delivery by AAV virus vectors expressing large genomes in hemophilia mouse and dog models: A strategy for broad clinical application. Mol. Ther.18, 1907-1916.
    • (2010) Mol. Ther. , vol.18 , pp. 1907-1916
    • Monahan, P.E.1    Lothrop, C.D.2    Sun, J.3
  • 47
    • 0036210202 scopus 로고    scopus 로고
    • Behavioral recovery in a primate model of Parkinson's disease by triple transduction of striatal cells with adeno-associated viral vectors expressing dopamine-synthesizing enzymes
    • Muramatsu, S., Fujimoto, K., Ikeguchi, K., et al. (2002). Behavioral recovery in a primate model of Parkinson's disease by triple transduction of striatal cells with adeno-associated viral vectors expressing dopamine-synthesizing enzymes. Hum. Gene Ther. 13, 345-354.
    • (2002) Hum. Gene Ther. , vol.13 , pp. 345-354
    • Muramatsu, S.1    Fujimoto, K.2    Ikeguchi, K.3
  • 48
    • 0034870596 scopus 로고    scopus 로고
    • Molecular genetics of macular degeneration
    • Musarella, M.A. (2001). Molecular genetics of macular degeneration. Doc. Ophthalmol. 102, 165-177.
    • (2001) Doc. Ophthalmol. , vol.102 , pp. 165-177
    • Musarella, M.A.1
  • 49
    • 34347246364 scopus 로고    scopus 로고
    • RNA interference-mediated suppression and replacement of human rhodopsin in vivo
    • O'Reilly, M., Palfi, A., Chadderton, N., et al. (2007). RNA interference-mediated suppression and replacement of human rhodopsin in vivo. Am. J. Hum. Genet. 81, 127-135.
    • (2007) Am. J. Hum. Genet. , vol.81 , pp. 127-135
    • O'Reilly, M.1    Palfi, A.2    Chadderton, N.3
  • 50
    • 33645224383 scopus 로고    scopus 로고
    • RNAi-based suppression and replacement of rds-peripherin in retinal orga-notypic culture
    • Palfi, A., Ader, M., Kiang, A.S., et al. (2006). RNAi-based suppression and replacement of rds-peripherin in retinal orga-notypic culture. Hum. Mutat. 27, 260-268.
    • (2006) Hum. Mutat. , vol.27 , pp. 260-268
    • Palfi, A.1    Ader, M.2    Kiang, A.S.3
  • 51
    • 77949733904 scopus 로고    scopus 로고
    • AAV-mediated rhodopsin replacement provides therapeutic benefit in mice with a targeted disruption of the rhodopsin gene
    • Palfi, A., Millington-Ward, S., Chadderton, N., et al. (2010). AAV-mediated rhodopsin replacement provides therapeutic benefit in mice with a targeted disruption of the rhodopsin gene. Hum. Gene Ther. 21, 311-323.
    • (2010) Hum. Gene Ther. , vol.21 , pp. 311-323
    • Palfi, A.1    Millington-Ward, S.2    Chadderton, N.3
  • 52
    • 27244446791 scopus 로고    scopus 로고
    • Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP
    • Pawlyk, B.S., Smith, A.J., Buch, P.K., et al. (2005). Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP. Invest. Ophthalmol. Vis. Sci. 46, 3039-3045.
    • (2005) Invest. Ophthalmol. Vis. Sci. , vol.46 , pp. 3039-3045
    • Pawlyk, B.S.1    Smith, A.J.2    Buch, P.K.3
  • 53
    • 19944430177 scopus 로고    scopus 로고
    • Biodistribution of rAAV vectors following intraocular administration: Evidence for the presence and persistence of vector DNA in the optic nerve and in the brain
    • Provost, N., Le Meur, G., Weber, M., et al. (2005). Biodistribution of rAAV vectors following intraocular administration: Evidence for the presence and persistence of vector DNA in the optic nerve and in the brain. Mol. Ther. 11, 275-283.
    • (2005) Mol. Ther. , vol.11 , pp. 275-283
    • Provost, N.1    Le Meur, G.2    Weber, M.3
  • 54
    • 12244311258 scopus 로고    scopus 로고
    • Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy
    • Reich, S.J., Auricchio, A., Hildinger, M., et al. (2003). Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy. Hum. Gene Ther. 14, 37-44.
    • (2003) Hum. Gene Ther. , vol.14 , pp. 37-44
    • Reich, S.J.1    Auricchio, A.2    Hildinger, M.3
  • 55
    • 42549166922 scopus 로고    scopus 로고
    • Subretinal delivery of recombinant AAV serotype 8 vector in dogs results in gene transfer to neurons in the brain
    • Stieger, K., Colle, M.A., Dubreil, L., et al. (2008). Subretinal delivery of recombinant AAV serotype 8 vector in dogs results in gene transfer to neurons in the brain. Mol. Ther. 16, 916-923.
    • (2008) Mol. Ther. , vol.16 , pp. 916-923
    • Stieger, K.1    Colle, M.A.2    Dubreil, L.3
  • 56
    • 61649101217 scopus 로고    scopus 로고
    • Detection of intact rAAV particles up to 6 years after successful gene transfer in the retina of dogs and primates
    • Stieger, K., Schroeder, J., Provost, N., et al. (2009). Detection of intact rAAV particles up to 6 years after successful gene transfer in the retina of dogs and primates. Mol. Ther. 17, 516-523.
    • (2009) Mol. Ther. , vol.17 , pp. 516-523
    • Stieger, K.1    Schroeder, J.2    Provost, N.3
  • 57
    • 74049085498 scopus 로고    scopus 로고
    • Effect of genome size on AAV vector packaging
    • Wu, Z., Yang, H., and Colosi, P. (2010). Effect of genome size on AAV vector packaging. Mol. Ther. 18, 80-86.
    • (2010) Mol. Ther. , vol.18 , pp. 80-86
    • Wu, Z.1    Yang, H.2    Colosi, P.3
  • 58
    • 77954161995 scopus 로고    scopus 로고
    • Genetics and pathological mechanisms of Usher syndrome
    • Yan, D., and Liu, X.Z. (2010). Genetics and pathological mechanisms of Usher syndrome. J. Hum. Genet. 55, 327-335.
    • (2010) J. Hum. Genet. , Issue.55 , pp. 327-335
    • Yan, D.1    Liu, X.Z.2
  • 59
    • 0034612248 scopus 로고    scopus 로고
    • Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy
    • Yan, Z., Zhang, Y., Duan, D., and Engelhardt, J.F. (2000). Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy. Proc. Natl. Acad. Sci. U.S.A. 97, 6716-6721.
    • (2000) Proc. Natl. Acad. Sci. U.S.A. , vol.97 , pp. 6716-6721
    • Yan, Z.1    Zhang, Y.2    Duan, D.3    Engelhardt, J.F.4
  • 60
    • 0036310297 scopus 로고    scopus 로고
    • Virus-mediated transduction of murine retina with adeno-associated virus: Effects of viral capsid and genome size
    • Yang, G.S., Schmidt, M., Yan, Z., et al. (2002). Virus-mediated transduction of murine retina with adeno-associated virus: Effects of viral capsid and genome size. J. Virol. 76, 7651-7660.
    • (2002) J. Virol. , vol.76 , pp. 7651-7660
    • Yang, G.S.1    Schmidt, M.2    Yan, Z.3
  • 61
    • 67349127313 scopus 로고    scopus 로고
    • Functional cone rescue by RdCVF protein in a dominant model of retinitis pigmentosa
    • Yang, Y., Mohand-Said, S., Danan, A., et al. (2009). Functional cone rescue by RdCVF protein in a dominant model of retinitis pigmentosa. Mol. Ther. 17, 787-795.
    • (2009) Mol. Ther. , vol.17 , pp. 787-795
    • Yang, Y.1    Mohand-Said, S.2    Danan, A.3
  • 62
    • 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, C.S., et al. (2008). Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc. Natl. Acad. Sci. U.S.A. 105, 7827-7832.
    • (2008) Proc. Natl. Acad. Sci. U.S.A. , vol.105 , pp. 7827-7832
    • Zhong, L.1    Li, B.2    Mah, C.S.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.