메뉴 건너뛰기




Volumn 19, Issue 2, 2011, Pages 234-242

Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa

Author keywords

[No Author keywords available]

Indexed keywords

CYCLIC GMP PHOSPHODIESTERASE; PARVOVIRUS VECTOR;

EID: 79551620165     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.273     Document Type: Article
Times cited : (126)

References (45)
  • 1
    • 0028939390 scopus 로고
    • Mutation spectrum of the gene encoding the subunit of rod phosphodiesterase among patients with autosomal recessive retinitis pigmentosa
    • McLaughlin, ME, Ehrhart, TL, Berson, EL and Dryja, TP (1995). Mutation spectrum of the gene encoding the subunit of rod phosphodiesterase among patients with autosomal recessive retinitis pigmentosa. Proc Natl Acad Sci USA 92: 3249-3253.
    • (1995) Proc Natl Acad Sci USA , vol.92 , pp. 3249-3253
    • McLaughlin, M.E.1    Ehrhart, T.L.2    Berson, E.L.3    Dryja, T.P.4
  • 3
    • 0027270053 scopus 로고
    • Recessive mutations in the gene encoding the -subunit of rod phosphodiesterase in patients with retinitis pigmentosa
    • McLaughlin, ME, Sandberg, MA, Berson, EL and Dryja, TP (1993). Recessive mutations in the gene encoding the -subunit of rod phosphodiesterase in patients with retinitis pigmentosa. Nat Genet 4: 130-134.
    • (1993) Nat Genet , vol.4 , pp. 130-134
    • McLaughlin, M.E.1    Sandberg, M.A.2    Berson, E.L.3    Dryja, T.P.4
  • 4
    • 0029893012 scopus 로고    scopus 로고
    • Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy
    • Bennett, J, Tanabe, T, Sun, D, Zeng, Y, Kjeldbye, H, Gouras, P et al. (1996). Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy. Nat Med 2: 649-654.
    • (1996) Nat Med , vol.2 , pp. 649-654
    • Bennett, J.1    Tanabe, T.2    Sun, D.3    Zeng, Y.4    Kjeldbye, H.5    Gouras, P.6
  • 5
    • 0030759730 scopus 로고    scopus 로고
    • Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration
    • Jomary, C, Vincent, KA, Grist, J, Neal, MJ and Jones, SE (1997). Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration. Gene Ther 4: 683-690.
    • (1997) Gene Ther , vol.4 , pp. 683-690
    • Jomary, C.1    Vincent, K.A.2    Grist, J.3    Neal, M.J.4    Jones, S.E.5
  • 6
    • 0032816002 scopus 로고    scopus 로고
    • Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer
    • Takahashi, M, Miyoshi, H, Verma, IM and Gage, FH (1999). Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer. J Virol 73: 7812-7816.
    • (1999) J Virol , vol.73 , pp. 7812-7816
    • Takahashi, M.1    Miyoshi, H.2    Verma, I.M.3    Gage, F.H.4
  • 8
    • 33846126652 scopus 로고    scopus 로고
    • Two mouse retinal degenerations caused by missense mutations in the -subunit of rod cGMP phosphodiesterase gene
    • Chang, B, Hawes, NL, Pardue, MT, German, AM, Hurd, RE, Davisson, MT et al. (2007). Two mouse retinal degenerations caused by missense mutations in the -subunit of rod cGMP phosphodiesterase gene. Vision Res 47: 624-633.
    • (2007) Vision Res , vol.47 , pp. 624-633
    • Chang, B.1    Hawes, N.L.2    Pardue, M.T.3    German, A.M.4    Hurd, R.E.5    Davisson, M.T.6
  • 9
    • 33845592141 scopus 로고    scopus 로고
    • Retinal organization in the retinal degeneration 10 (rd10) mutant mouse: A morphological and ERG study
    • Gargini, C, Terzibasi, E, Mazzoni, F and Strettoi, E (2007). Retinal organization in the retinal degeneration 10 (rd10) mutant mouse: a morphological and ERG study. J Comp Neurol 500: 222-238.
    • (2007) J Comp Neurol , vol.500 , pp. 222-238
    • Gargini, C.1    Terzibasi, E.2    Mazzoni, F.3    Strettoi, E.4
  • 10
    • 0036662776 scopus 로고    scopus 로고
    • Morphological and functional abnormalities in the inner retina of the rd/rd mouse
    • Strettoi, E, Porciatti, V, Falsini, B, Pignatelli, V and Rossi, C (2002). Morphological and functional abnormalities in the inner retina of the rd/rd mouse. J Neurosci 22: 5492-5504.
    • (2002) J Neurosci , vol.22 , pp. 5492-5504
    • Strettoi, E.1    Porciatti, V.2    Falsini, B.3    Pignatelli, V.4    Rossi, C.5
  • 11
    • 0037381093 scopus 로고    scopus 로고
    • Remodeling of second-order neurons in the retina of rd/rd mutant mice
    • Strettoi, E, Pignatelli, V, Rossi, C, Porciatti, V and Falsini, B (2003). Remodeling of second-order neurons in the retina of rd/rd mutant mice. Vision Res 43: 867-877.
    • (2003) Vision Res , vol.43 , pp. 867-877
    • Strettoi, E.1    Pignatelli, V.2    Rossi, C.3    Porciatti, V.4    Falsini, B.5
  • 13
    • 0033166072 scopus 로고    scopus 로고
    • Retinal rod photoreceptor-specific gene mutation perturbs cone pathway development
    • Banin, E, Cideciyan, AV, Alemán, TS, Petters, RM, Wong, F, Milam, AH et al. (1999). Retinal rod photoreceptor-specific gene mutation perturbs cone pathway development. Neuron 23: 549-557.
    • (1999) Neuron , vol.23 , pp. 549-557
    • Banin, E.1    Cideciyan, A.V.2    Alemán, T.S.3    Petters, R.M.4    Wong, F.5    Milam, A.H.6
  • 15
    • 44649189096 scopus 로고    scopus 로고
    • Extreme retinal remodeling triggered by light damage: Implications for age related macular degeneration
    • Marc, RE, Jones, BW, Watt, CB, Vazquez-Chona, F, Vaughan, DK and Organisciak, DT (2008). Extreme retinal remodeling triggered by light damage: implications for age related macular degeneration. Mol Vis 14: 782-806.
    • (2008) Mol Vis , vol.14 , pp. 782-806
    • Marc, R.E.1    Jones, B.W.2    Watt, C.B.3    Vazquez-Chona, F.4    Vaughan, D.K.5    Organisciak, D.T.6
  • 16
    • 0032052472 scopus 로고    scopus 로고
    • Histopathology of the human retina in retinitis pigmentosa
    • Milam, AH, Li, ZY and Fariss, RN (1998). Histopathology of the human retina in retinitis pigmentosa. Prog Retin Eye Res 17: 175-205.
    • (1998) Prog Retin Eye Res , vol.17 , pp. 175-205
    • Milam, A.H.1    Li, Z.Y.2    Fariss, R.N.3
  • 18
    • 0942298092 scopus 로고    scopus 로고
    • Inner retinal abnormalities in a mouse model of Leber's congenital amaurosis
    • Pignatelli, V, Cepko, CL and Strettoi, E (2004). Inner retinal abnormalities in a mouse model of Leber's congenital amaurosis. J Comp Neurol 469: 351-359.
    • (2004) J Comp Neurol , vol.469 , pp. 351-359
    • Pignatelli, V.1    Cepko, C.L.2    Strettoi, E.3
  • 19
    • 79551639479 scopus 로고    scopus 로고
    • Functional changes in inner retinal neurons in animal models of photoreceptor degeneration
    • Puthussery, T and Taylor, WR (2010). Functional changes in inner retinal neurons in animal models of photoreceptor degeneration. Adv Exp Med Biol 664: 525-532.
    • (2010) Adv Exp Med Biol , vol.664 , pp. 525-532
    • Puthussery, T.1    Taylor, W.R.2
  • 20
    • 0037381299 scopus 로고    scopus 로고
    • Functional modifications in rod bipolar cells in a mouse model of retinitis pigmentosa
    • Varela, C, Igartua, I, De la Rosa, EJ and De la Villa, P (2003). Functional modifications in rod bipolar cells in a mouse model of retinitis pigmentosa. Vision Res 43: 879-885.
    • (2003) Vision Res , vol.43 , pp. 879-885
    • Varela, C.1    Igartua, I.2    De La Rosa, E.J.3    De La Villa, P.4
  • 21
    • 0034718480 scopus 로고    scopus 로고
    • Modifications of retinal neurons in a mouse model of retinitis pigmentosa
    • Strettoi, E and Pignatelli, V (2000). Modifications of retinal neurons in a mouse model of retinitis pigmentosa. Proc Natl Acad Sci USA 97: 11020-11025.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 11020-11025
    • Strettoi, E.1    Pignatelli, V.2
  • 22
    • 46749111426 scopus 로고    scopus 로고
    • Functional stability of retinal ganglion cells after degeneration-induced changes in synaptic input
    • Margolis, DJ, Newkirk, G, Euler, T and Detwiler, PB (2008). Functional stability of retinal ganglion cells after degeneration-induced changes in synaptic input. J Neurosci 28: 6526-6536.
    • (2008) J Neurosci , vol.28 , pp. 6526-6536
    • Margolis, D.J.1    Newkirk, G.2    Euler, T.3    Detwiler, P.B.4
  • 23
    • 58149385767 scopus 로고    scopus 로고
    • Retinal ganglion cells survive and maintain normal dendritic morphology in a mouse model of inherited photoreceptor degeneration
    • Mazzoni, F, Novelli, E and Strettoi, E (2008). Retinal ganglion cells survive and maintain normal dendritic morphology in a mouse model of inherited photoreceptor degeneration. J Neurosci 28: 14282-14292.
    • (2008) J Neurosci , vol.28 , pp. 14282-14292
    • Mazzoni, F.1    Novelli, E.2    Strettoi, E.3
  • 24
    • 40949154259 scopus 로고    scopus 로고
    • Emergence of sustained spontaneous hyperactivity and temporary preservation of off responses in ganglion cells of the retinal degeneration (rd1) mouse
    • Stasheff, SF (2008). Emergence of sustained spontaneous hyperactivity and temporary preservation of OFF responses in ganglion cells of the retinal degeneration (rd1) mouse. J Neurophysiol 99: 1408-1421.
    • (2008) J Neurophysiol , vol.99 , pp. 1408-1421
    • Stasheff, S.F.1
  • 25
    • 53449093170 scopus 로고    scopus 로고
    • AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDE? mutation
    • Pang, JJ, Boye, SL, Kumar, A, Dinculescu, A, Deng, W, Li, J et al. (2008). AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDE? mutation. Invest Ophthalmol Vis Sci 49: 4278-4283.
    • (2008) Invest Ophthalmol Vis Sci , vol.49 , pp. 4278-4283
    • Pang, J.J.1    Boye, S.L.2    Kumar, A.3    Dinculescu, A.4    Deng, W.5    Li, J.6
  • 27
    • 9644268242 scopus 로고    scopus 로고
    • Rescue of retinal degeneration by intravitreally injected adult bone marrow-derived lineage-negative hematopoietic stem cells
    • Otani, A, Dorrell, MI, Kinder, K, Moreno, SK, Nusinowitz, S, Banin, E et al. (2004). Rescue of retinal degeneration by intravitreally injected adult bone marrow-derived lineage-negative hematopoietic stem cells. J Clin Invest 114: 765-774.
    • (2004) J Clin Invest , vol.114 , pp. 765-774
    • Otani, A.1    Dorrell, M.I.2    Kinder, K.3    Moreno, S.K.4    Nusinowitz, S.5    Banin, E.6
  • 28
    • 74349104948 scopus 로고    scopus 로고
    • Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations
    • Sun, X, Pawlyk, B, Xu, X, Liu, X, Bulgakov, OV, Adamian, M et al. (2010). Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations. Gene Ther 17: 117-131.
    • (2010) Gene Ther , vol.17 , pp. 117-131
    • Sun, X.1    Pawlyk, B.2    Xu, X.3    Liu, X.4    Bulgakov, O.V.5    Adamian, M.6
  • 29
    • 27244446791 scopus 로고    scopus 로고
    • Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP
    • Pawlyk, BS, Smith, AJ, Buch, PK, Adamian, M, Hong, DH, Sandberg, MA 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    Adamian, M.4    Hong, D.H.5    Sandberg, M.A.6
  • 30
    • 40249098715 scopus 로고    scopus 로고
    • Assessment of ocular transduction using single-stranded and selfcomplementary recombinant adeno-associated virus serotype 2/8
    • Natkunarajah, M, Trittibach, P, McIntosh, J, Duran, Y, Barker, SE, Smith, AJ et al. (2008). Assessment of ocular transduction using single-stranded and selfcomplementary recombinant adeno-associated virus serotype 2/8. Gene Ther 15: 463-467.
    • (2008) Gene Ther , vol.15 , pp. 463-467
    • Natkunarajah, M.1    Trittibach, P.2    McIntosh, J.3    Duran, Y.4    Barker, S.E.5    Smith, A.J.6
  • 31
    • 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, Li, B, Zolotukhin, S, Govindasamy, L et al. (2007). A dual role of EGFR protein tyrosine kinase signaling in ubiquitination of AAV2 capsids and viral second-strand DNA synthesis. Mol Ther 15: 1323-1330.
    • (2007) Mol Ther , vol.15 , pp. 1323-1330
    • Zhong, L.1    Zhao, W.2    Wu, J.3    Li, B.4    Zolotukhin, S.5    Govindasamy, L.6
  • 32
    • 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, Govindasamy, L, Agbandje-McKenna, M, Cooper, M 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 USA 105: 7827-7832.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 7827-7832
    • Zhong, L.1    Li, B.2    Mah, C.S.3    Govindasamy, L.4    Agbandje-Mckenna, M.5    Cooper, M.6
  • 33
    • 61649095399 scopus 로고    scopus 로고
    • Highefficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors
    • Petrs-Silva, H, Dinculescu, A, Li, Q, Min, SH, Chiodo, V, Pang, JJ et al. (2009). Highefficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther 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    Pang, J.J.6
  • 34
    • 79551628877 scopus 로고    scopus 로고
    • Reproducibility of spectral-domain optical coherence tomography total retinal thickness measurements in mice
    • epub ahead of print
    • Gabriele, ML, Ishikawa, H, Schuman, JS, Bilonick, RA, Kim, JS, Kagemann, L et al. (2010). Reproducibility of Spectral-Domain Optical Coherence Tomography Total Retinal Thickness Measurements in Mice. Invest Ophthalmol Vis Sci (epub ahead of print).
    • (2010) Invest Ophthalmol Vis Sci
    • Gabriele, M.L.1    Ishikawa, H.2    Schuman, J.S.3    Bilonick, R.A.4    Kim, J.S.5    Kagemann, L.6
  • 36
    • 34347327080 scopus 로고    scopus 로고
    • Long-term visual prognoses in patients with retinitis pigmentosa: The Ludwig von Sallmann lecture
    • Berson, EL (2007). Long-term visual prognoses in patients with retinitis pigmentosa: the Ludwig von Sallmann lecture. Exp Eye Res 85: 7-14.
    • (2007) Exp Eye Res , vol.85 , pp. 7-14
    • Berson, E.L.1
  • 37
    • 33645974269 scopus 로고    scopus 로고
    • Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration
    • Bi, A, Cui, J, Ma, YP, Olshevskaya, E, Pu, M, Dizhoor, AM et al. (2006). Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration. Neuron 50: 23-33.
    • (2006) Neuron , vol.50 , pp. 23-33
    • Bi, A.1    Cui, J.2    Ma, Y.P.3    Olshevskaya, E.4    Pu, M.5    Dizhoor, A.M.6
  • 38
    • 78149260595 scopus 로고    scopus 로고
    • Cone and rod photoreceptor transplantation in models of the childhood retinopathy Leber congenital amaurosis using flow-sorted Crx-positive donor cells
    • Lakowski, J, Baron, M, Bainbridge, J, Barber, AC, Pearson, RA, Ali, RR et al. (2010). Cone and rod photoreceptor transplantation in models of the childhood retinopathy Leber congenital amaurosis using flow-sorted Crx-positive donor cells. Hum Mol Genet 19: 4545-4559.
    • (2010) Hum Mol Genet , vol.19 , pp. 4545-4559
    • Lakowski, J.1    Baron, M.2    Bainbridge, J.3    Barber, A.C.4    Pearson, R.A.5    Ali, R.R.6
  • 39
    • 33749139130 scopus 로고    scopus 로고
    • Lightdriven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1
    • Haire, SE, Pang, J, Boye, SL, Sokal, I, Craft, CM, Palczewski, K et al. (2006). Lightdriven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1. Invest Ophthalmol Vis Sci 47: 3745-3753.
    • (2006) Invest Ophthalmol Vis Sci , vol.47 , pp. 3745-3753
    • Haire, S.E.1    Pang, J.2    Boye, S.L.3    Sokal, I.4    Craft, C.M.5    Palczewski, K.6
  • 40
    • 0034091924 scopus 로고    scopus 로고
    • Production and purification of recombinant adeno-associated virus
    • Hauswirth, WW, Lewin, AS, Zolotukhin, S and Muzyczka, N (2000). Production and purification of recombinant adeno-associated virus. Meth Enzymol 316: 743-761.
    • (2000) Meth Enzymol , vol.316 , pp. 743-761
    • Hauswirth, W.W.1    Lewin, A.S.2    Zolotukhin, S.3    Muzyczka, N.4
  • 41
    • 77956208538 scopus 로고    scopus 로고
    • Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse
    • Boye, SE, Boye, SL, Pang, J, Ryals, R, Everhart, D, Umino, Y et al. (2010). Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. PLoS ONE 5: e11306.
    • (2010) PLoS ONE , vol.5
    • Boye, S.E.1    Boye, S.L.2    Pang, J.3    Ryals, R.4    Everhart, D.5    Umino, Y.6
  • 42
    • 77954493492 scopus 로고    scopus 로고
    • Selfcomplementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency
    • Pang, J, Boye, SE, Lei, B, Boye, SL, Everhart, D, Ryals, R et al. (2010). Selfcomplementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency. Gene Ther 17: 815-826.
    • (2010) Gene Ther , vol.17 , pp. 815-826
    • Pang, J.1    Boye, S.E.2    Lei, B.3    Boye, S.L.4    Everhart, D.5    Ryals, R.6
  • 43
    • 38149022652 scopus 로고    scopus 로고
    • Speed, spatial, and temporal tuning of rod and cone vision in mouse
    • Umino, Y, Solessio, E and Barlow, RB (2008). Speed, spatial, and temporal tuning of rod and cone vision in mouse. J Neurosci 28: 189-198.
    • (2008) J Neurosci , vol.28 , pp. 189-198
    • Umino, Y.1    Solessio, E.2    Barlow, R.B.3
  • 44
    • 4344660019 scopus 로고    scopus 로고
    • Adenoviral-mediated gene transfer to retinal explants during development and degeneration
    • Pang, J, Cheng, M, Stevenson, D, Trousdale, MD, Dorey, CK and Blanks, JC (2004). Adenoviral-mediated gene transfer to retinal explants during development and degeneration. Exp Eye Res 79: 189-201.
    • (2004) Exp Eye Res , vol.79 , pp. 189-201
    • Pang, J.1    Cheng, M.2    Stevenson, D.3    Trousdale, M.D.4    Dorey, C.K.5    Blanks, J.C.6
  • 45
    • 20844450821 scopus 로고    scopus 로고
    • Retinal degeneration 12 (rd12) a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA)
    • Pang, JJ, Chang, B, Hawes, NL, Hurd, RE, Davisson, MT, Li, J et al. (2005). Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA). Mol Vis 11: 152-162.
    • (2005) Mol Vis , vol.11 , pp. 152-162
    • Pang, J.J.1    Chang, B.2    Hawes, N.L.3    Hurd, R.E.4    Davisson, M.T.5    Li, J.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.