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    Results: 1 to 20 of 114

    1.

    Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65-/- knockout mouse eye results in limited rescue.

    Lai CM, Yu MJ, Brankov M, Barnett NL, Zhou X, Redmond TM, Narfstrom K, Rakoczy PE.

    Genet Vaccines Ther. 2004 Apr 27;2(1):3.PMID: 15109394 [PubMed - as supplied by publisher]Related articlesFree article

    2.

    Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis.

    Bemelmans AP, Kostic C, Crippa SV, Hauswirth WW, Lem J, Munier FL, Seeliger MW, Wenzel A, Arsenijevic Y.

    PLoS Med. 2006 Oct;3(10):e347.PMID: 17032058 [PubMed - indexed for MEDLINE]Related articlesFree article

    3.

    Gene therapeutic prospects in early onset of severe retinal dystrophy: restoration of vision in RPE65 Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium.

    Rolling F, Le Meur G, Stieger K, Smith AJ, Weber M, Deschamps JY, Nivard D, Mendes-Madeira A, Provost N, Péréon Y, Cherel Y, Ali RR, Hamel C, Moullier P, Rolling F.

    Bull Mem Acad R Med Belg. 2006;161(10-12):497-508; discussion 508-9.PMID: 17503728 [PubMed - indexed for MEDLINE]Related articles

    4.

    Bax-induced apoptosis in Leber's congenital amaurosis: a dual role in rod and cone degeneration.

    Hamann S, Schorderet DF, Cottet S.

    PLoS One. 2009 Aug 12;4(8):e6616.PMID: 19672311 [PubMed - indexed for MEDLINE]Related articlesFree article

    5.

    Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.

    Narfström K, Katz ML, Bragadottir R, Seeliger M, Boulanger A, Redmond TM, Caro L, Lai CM, Rakoczy PE.

    Invest Ophthalmol Vis Sci. 2003 Apr;44(4):1663-72.PMID: 12657607 [PubMed - indexed for MEDLINE]Related articlesFree article

    6.

    Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye.

    Li W, Kong F, Li X, Dai X, Liu X, Zheng Q, Wu R, Zhou X, Lü F, Chang B, Li Q, Hauswirth WW, Qu J, Pang JJ.

    Mol Vis. 2009;15:267-75. Epub 2009 Feb 6.PMID: 19190735 [PubMed - indexed for MEDLINE]Related articlesFree article

    7.

    Postsurgical assessment and long-term safety of recombinant adeno-associated virus-mediated gene transfer into the retinas of dogs and primates.

    Le Meur G, Weber M, Péréon Y, Mendes-Madeira A, Nivard D, Deschamps JY, Moullier P, Rolling F.

    Arch Ophthalmol. 2005 Apr;123(4):500-6.PMID: 15824224 [PubMed - indexed for MEDLINE]Related articles

    8.

    Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium.

    Le Meur G, Stieger K, Smith AJ, Weber M, Deschamps JY, Nivard D, Mendes-Madeira A, Provost N, Péréon Y, Cherel Y, Ali RR, Hamel C, Moullier P, Rolling F.

    Gene Ther. 2007 Feb;14(4):292-303. Epub 2006 Oct 5.PMID: 17024105 [PubMed - indexed for MEDLINE]Related articles

    9.

    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, Noorwez SM, Malhotra R, McDowell JH, Kaushal S, Hauswirth WW, Nusinowitz S, Thompson DA, Heckenlively JR.

    Mol Vis. 2005 Feb 28;11:152-62.PMID: 15765048 [PubMed - indexed for MEDLINE]Related articlesFree article

    10.

    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, Bennicelli J, Dejneka NS, Pearce-Kelling SE, Maguire AM, Palczewski K, Hauswirth WW, Jacobson SG.

    Mol Ther. 2005 Dec;12(6):1072-82. Epub 2005 Oct 14.PMID: 16226919 [PubMed - indexed for MEDLINE]Related articles

    11.

    Effects of adeno-associated virus-vectored ciliary neurotrophic factor on retinal structure and function in mice with a P216L rds/peripherin mutation.

    Bok D, Yasumura D, Matthes MT, Ruiz A, Duncan JL, Chappelow AV, Zolutukhin S, Hauswirth W, LaVail MM.

    Exp Eye Res. 2002 Jun;74(6):719-35.PMID: 12126945 [PubMed - indexed for MEDLINE]Related articles

    12.

    Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina.

    Bainbridge JW, Mistry A, Schlichtenbrede FC, Smith A, Broderick C, De Alwis M, Georgiadis A, Taylor PM, Squires M, Sethi C, Charteris D, Thrasher AJ, Sargan D, Ali RR.

    Gene Ther. 2003 Aug;10(16):1336-44.PMID: 12883530 [PubMed - indexed for MEDLINE]Related articles

    13.

    RPE65 gene delivery restores isomerohydrolase activity and prevents early cone loss in Rpe65-/- mice.

    Chen Y, Moiseyev G, Takahashi Y, Ma JX.

    Invest Ophthalmol Vis Sci. 2006 Mar;47(3):1177-84.PMID: 16505056 [PubMed - indexed for MEDLINE]Related articlesFree article

    14.

    Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis.

    Roman AJ, Boye SL, Aleman TS, Pang JJ, McDowell JH, Boye SE, Cideciyan AV, Jacobson SG, Hauswirth WW.

    Mol Vis. 2007 Sep 18;13:1701-10.PMID: 17960108 [PubMed - indexed for MEDLINE]Related articlesFree article

    15.

    In vivo gene therapy in young and adult RPE65-/- dogs produces long-term visual improvement.

    Narfström K, Katz ML, Ford M, Redmond TM, Rakoczy E, Bragadóttir R.

    J Hered. 2003 Jan-Feb;94(1):31-7.PMID: 12692160 [PubMed - indexed for MEDLINE]Related articlesFree article

    16.

    Downregulation of cone-specific gene expression and degeneration of cone photoreceptors in the Rpe65-/- mouse at early ages.

    Znoiko SL, Rohrer B, Lu K, Lohr HR, Crouch RK, Ma JX.

    Invest Ophthalmol Vis Sci. 2005 Apr;46(4):1473-9.PMID: 15790918 [PubMed - indexed for MEDLINE]Related articlesFree article

    17.

    Synaptic pathology in retinoschisis knockout (Rs1-/y) mouse retina and modification by rAAV-Rs1 gene delivery.

    Takada Y, Vijayasarathy C, Zeng Y, Kjellstrom S, Bush RA, Sieving PA.

    Invest Ophthalmol Vis Sci. 2008 Aug;49(8):3677-86.PMID: 18660429 [PubMed - indexed for MEDLINE]Related articlesFree article

    18.

    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, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG.

    Hum Gene Ther. 2008 Oct;19(10):979-90.PMID: 18774912 [PubMed - indexed for MEDLINE]Related articles

    19.

    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, Ali RR, Li T.

    Invest Ophthalmol Vis Sci. 2005 Sep;46(9):3039-45.PMID: 16123399 [PubMed - indexed for MEDLINE]Related articlesFree article

    20.

    Nrl-knockout mice deficient in Rpe65 fail to synthesize 11-cis retinal and cone outer segments.

    Feathers KL, Lyubarsky AL, Khan NW, Teofilo K, Swaroop A, Williams DS, Pugh EN Jr, Thompson DA.

    Invest Ophthalmol Vis Sci. 2008 Mar;49(3):1126-35.PMID: 18326740 [PubMed - indexed for MEDLINE]Related articlesFree article

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