메뉴 건너뛰기




Volumn 664, Issue , 2010, Pages 611-619

Gene therapy in the retinal degeneration slow model of retinitis pigmentosa

Author keywords

[No Author keywords available]

Indexed keywords

MEMBRANE PROTEIN; RETINA DEGENERATION SLOW PROTEIN; UNCLASSIFIED DRUG;

EID: 79959619541     PISSN: 00652598     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-4419-1399-9_70     Document Type: Conference Paper
Times cited : (10)

References (47)
  • 9
    • 46549086021 scopus 로고    scopus 로고
    • The spectrum of retinal dystrophies caused by mutations in the peripherin/RDS gene
    • Boon CJ, den Hollander AI, Hoyng CB et al (2008) The spectrum of retinal dystrophies caused by mutations in the peripherin/RDS gene. Prog Retin Eye Res 27:213-235
    • (2008) Prog Retin Eye Res , vol.27 , pp. 213-235
    • Boon, C.J.1    Den Hollander, A.I.2    Hoyng, C.B.3
  • 10
    • 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
    • DOI 10.1016/j.ymthe.2006.05.019, PII S1525001606002292
    • Buch PK, MacLaren RE, Duran 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 (Pubitemid 44480755)
    • (2006) Molecular Therapy , vol.14 , Issue.5 , pp. 700-709
    • Buch, P.K.1    MacLaren, R.E.2    Duran, Y.3    Balaggan, K.S.4    MacNeil, A.5    Schlichtenbrede, F.C.6    Smith, A.J.7    Ali, R.R.8
  • 12
    • 0032533962 scopus 로고    scopus 로고
    • Intraocular gene transfer of ciliary neurotrophic factor prevents death and increases responsiveness of rod photoreceptors in the retinal degeneration slow mouse
    • Cayouette M, Behn D, Sendtner M et al (1998) Intraocular gene transfer of ciliary neurotrophic factor prevents death and increases responsiveness of rod photoreceptors in the retinal degeneration slow mouse. J Neurosci 18:9282-9293 (Pubitemid 28512459)
    • (1998) Journal of Neuroscience , vol.18 , Issue.22 , pp. 9282-9293
    • Cayouette, M.1    Behn, D.2    Sendtner, M.3    Lachapelle, P.4    Gravel, C.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 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
  • 16
    • 38549148792 scopus 로고    scopus 로고
    • Late-onset cone photoreceptor degeneration induced by R172W mutation in Rds and partial rescue by gene supplementation
    • DOI 10.1167/iovs.07-0663
    • Conley S, Nour M, Fliesler SJ et al (2007) Late-onset cone photoreceptor degeneration induced by R172W mutation in Rds and partial rescue by gene supplementation. Invest Ophthalmol Vis Sci 48:5397-5407 (Pubitemid 351260838)
    • (2007) Investigative Ophthalmology and Visual Science , vol.48 , Issue.12 , pp. 5397-5407
    • Conley, S.1    Nour, M.2    Fliesler, S.J.3    Naash, M.I.4
  • 18
    • 44049090443 scopus 로고    scopus 로고
    • AAV vectors for RNA-based modulation of gene expression
    • DOI 10.1038/gt.2008.69, PII GT200869, Special Issue: AAV Vectors for Clinical GeneTherapy
    • Danos O (2008) AAV vectors for RNA-based modulation of gene expression. Gene Ther 15: 864-869 (Pubitemid 351712620)
    • (2008) Gene Therapy , vol.15 , Issue.11 , pp. 864-869
    • Danos, O.1
  • 20
    • 33845510734 scopus 로고    scopus 로고
    • The role of Rds in outer segment morphogenesis and human retinal disease
    • DOI 10.1080/13816810600976806, PII NK55137435692077
    • Farjo R, Naash MI (2006) The role of Rds in outer segment morphogenesis and human retinal disease. Ophthalmic Genet 27:117-122 (Pubitemid 44915774)
    • (2006) Ophthalmic Genetics , vol.27 , Issue.4 , pp. 117-122
    • Farjo, R.1    Naash, M.I.2
  • 21
    • 33645748724 scopus 로고    scopus 로고
    • Retention of function without normal disc morphogenesis occurs in cone but not rod photoreceptors
    • Farjo R, Skaggs JS, Nagel BA et al (2006b) Retention of function without normal disc morphogenesis occurs in cone but not rod photoreceptors. J Cell Biol 173:59-68
    • (2006) J Cell Biol , vol.173 , pp. 59-68
    • Farjo, R.1    Skaggs, J.S.2    Nagel, B.A.3
  • 22
    • 34248570405 scopus 로고    scopus 로고
    • Efficient non-viral ocular gene transfer with compacted DNA nanoparticles
    • Farjo R, Skaggs J, Quiambao AB et al (2006a) Efficient non-viral ocular gene transfer with compacted DNA nanoparticles. PLoS ONE 1:e38
    • (2006) PLoS ONE , vol.1
    • Farjo, R.1    Skaggs, J.2    Quiambao, A.B.3
  • 23
    • 0036500230 scopus 로고    scopus 로고
    • On the genetics of retinitis pigmentosa and on mutation-independent approaches to therapeutic intervention
    • DOI 10.1093/emboj/21.5.857
    • Farrar GJ, Kenna PF, Humphries P (2002) On the genetics of retinitis pigmentosa and on mutationindependent approaches to therapeutic intervention. EMBO J 21:857-864 (Pubitemid 34206159)
    • (2002) EMBO Journal , vol.21 , Issue.5 , pp. 857-864
    • Farrar, G.J.1    Kenna, P.F.2    Humphries, P.3
  • 25
    • 33751232603 scopus 로고    scopus 로고
    • Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme
    • DOI 10.1016/j.exer.2006.08.014, PII S0014483506003666
    • Gorbatyuk M, Justilien V, Liu J et al (2007a) Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme. Exp Eye Res 84:44-52 (Pubitemid 44792051)
    • (2007) Experimental Eye Research , vol.84 , Issue.1 , pp. 44-52
    • Gorbatyuk, M.1    Justilien, V.2    Liu, J.3    Hauswirth, W.W.4    Lewin, A.S.5
  • 26
    • 33947595161 scopus 로고    scopus 로고
    • Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery
    • DOI 10.1016/j.visres.2006.11.026, PII S004269890600558X
    • Gorbatyuk M, Justilien V, Liu J et al (2007b) Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery. Vis Res 47:1202-1208 (Pubitemid 46482623)
    • (2007) Vision Research , vol.47 , Issue.9 , pp. 1202-1208
    • Gorbatyuk, M.1    Justilien, V.2    Liu, J.3    Hauswirth, W.W.4    Lewin, A.S.5
  • 28
    • 0033862086 scopus 로고    scopus 로고
    • Ribozyme uses in retinal gene therapy
    • DOI 10.1016/S1350-9462(00)00007-0, PII S1350946200000070
    • Hauswirth WW, Lewin AS (2000) Ribozyme uses in retinal gene therapy. Prog Retin Eye Res 19:689-710 (Pubitemid 30616360)
    • (2000) Progress in Retinal and Eye Research , vol.19 , Issue.6 , pp. 689-710
    • Hauswirth, W.W.1    Lewin, A.S.2
  • 29
    • 0025720710 scopus 로고
    • Mutations in the human retinal degeneration slow gene in autosomal dominant retinitis pigmentosa
    • Kajiwara K, Hahn LB, Mukai S et al (1991) Mutations in the human retinal degeneration slow gene in autosomal dominant retinitis pigmentosa. Nature 354:480-483 (Pubitemid 21896864)
    • (1991) Nature , vol.354 , Issue.6353 , pp. 480-483
    • Kajiwara, K.1    Hahn, L.B.2    Mukai, S.3    Travis, G.H.4    Berson, E.L.5    Dryja, T.P.6
  • 33
    • 0034633619 scopus 로고    scopus 로고
    • Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late-stage therapy
    • LaVail MM, Yasumura D, Matthes MT et al (2000) Ribozyme rescue of photoreceptor cells in P23H transgenic rats: Long-term survival and late-stage therapy. Proc Natl Acad Sci U S A 97:11488-11493
    • (2000) Proc Natl Acad Sci U S A , vol.97 , pp. 11488-11493
    • LaVail, M.M.1    Yasumura, D.2    Matthes, M.T.3
  • 37
    • 44049094771 scopus 로고    scopus 로고
    • Clinical gene therapy using recombinant adeno-associated virus vectors
    • DOI 10.1038/gt.2008.68, PII GT200868, Special Issue: AAV Vectors for Clinical GeneTherapy
    • Mueller C, Flotte TR (2008) Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther 15:858-863 (Pubitemid 351712619)
    • (2008) Gene Therapy , vol.15 , Issue.11 , pp. 858-863
    • Mueller, C.1    Flotte, T.R.2
  • 39
    • 38949131913 scopus 로고    scopus 로고
    • Genetic supplementation of RDS alleviates a loss-offunction phenotype in C214S model of retinitis pigmentosa
    • Nour M, Fliesler SJ, Naash MI (2008) Genetic supplementation of RDS alleviates a loss-offunction phenotype in C214S model of retinitis pigmentosa. Adv Exp Med Biol 613:129-138
    • (2008) Adv Exp Med Biol , vol.613 , pp. 129-138
    • Nour, M.1    Fliesler, S.J.2    Naash, M.I.3
  • 40
    • 63949086607 scopus 로고    scopus 로고
    • A novel mutation in Prph2, a gene regulated by Nr2e3, causes retinal degeneration and outer-segment defects similar to Nr2e3 (rd7/rd7) retinas
    • Nystuen AM, Sachs AJ, Yuan Y et al (2008) A novel mutation in Prph2, a gene regulated by Nr2e3, causes retinal degeneration and outer-segment defects similar to Nr2e3 (rd7/rd7) retinas. Mamm Genome 9:623-633
    • (2008) Mamm Genome , vol.9 , pp. 623-633
    • Nystuen, A.M.1    Sachs, A.J.2    Yuan, Y.3
  • 43
    • 0035504663 scopus 로고    scopus 로고
    • Gene replacement therapy in the retinal degeneration slow (rds) mouse: The effect on retinal degeneration following partial transduction of the retina
    • Sarra GM, Stephens C, de Alwis M et al (2001) Gene replacement therapy in the retinal degeneration slow (rds) mouse: The effect on retinal degeneration following partial transduction of the retina. Hum Mol Genet 10:2353-2361 (Pubitemid 33069541)
    • (2001) Human Molecular Genetics , vol.10 , Issue.21 , pp. 2353-2361
    • Sarra, G.-M.1    Stephens, C.2    De Alwis, M.3    Bainbridge, J.W.B.4    Smith, A.J.5    Thrasher, A.J.6    Ali, R.R.7
  • 46
    • 20544441047 scopus 로고    scopus 로고
    • 214 Ser mutation in peripherin/rds causes a loss-of-function phenotype in transgenic mice
    • DOI 10.1042/BJ20041960
    • Stricker HM, Ding XQ, Quiambao A et al (2005) The Cys214→Ser mutation in peripherin/rds causes a loss-of-function phenotype in transgenic mice. Biochem J 388:605-613 (Pubitemid 40839938)
    • (2005) Biochemical Journal , vol.388 , Issue.2 , pp. 605-613
    • Stricker, H.M.1    Ding, X.-Q.2    Quiambao, A.3    Fliesler, S.J.4    Naash, M.I.5
  • 47
    • 0018174467 scopus 로고
    • A new H-2-linked mutation, rds, causing retinal degeneration in the mouse
    • van Nie R, Ivanyi D, Demant P (1978) A new H-2-linked mutation, rds, causing retinal degeneration in the mouse. Tissue Antigens 12:106-108 (Pubitemid 9002921)
    • (1978) Tissue Antigens , vol.12 , Issue.2 , pp. 106-108
    • Van Nie, R.1    Ivanyi, D.2    Demant, P.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.