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Journal Abstract Search

319 related items for PubMed ID: 17960108

  • 1. 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. PubMed ID: 17960108
    [Abstract] [Full Text] [Related]

  • 2. 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. PubMed ID: 15765048
    [Abstract] [Full Text] [Related]

  • 3. Retinal disease in Rpe65-deficient mice: comparison to human leber congenital amaurosis due to RPE65 mutations.
    Caruso RC, Aleman TS, Cideciyan AV, Roman AJ, Sumaroka A, Mullins CL, Boye SL, Hauswirth WW, Jacobson SG.
    Invest Ophthalmol Vis Sci; 2010 Oct 28; 51(10):5304-13. PubMed ID: 20484585
    [Abstract] [Full Text] [Related]

  • 4. Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis.
    Pang JJ, Chang B, Kumar A, Nusinowitz S, Noorwez SM, Li J, Rani A, Foster TC, Chiodo VA, Doyle T, Li H, Malhotra R, Teusner JT, McDowell JH, Min SH, Li Q, Kaushal S, Hauswirth WW.
    Mol Ther; 2006 Mar 28; 13(3):565-72. PubMed ID: 16223604
    [Abstract] [Full Text] [Related]

  • 5. 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 Mar 28; 161(10-12):497-508; discussion 508-9. PubMed ID: 17503728
    [Abstract] [Full Text] [Related]

  • 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 Mar 28; 15():267-75. PubMed ID: 19190735
    [Abstract] [Full Text] [Related]

  • 7. 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 28; 46(9):3039-45. PubMed ID: 16123399
    [Abstract] [Full Text] [Related]

  • 8. Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection.
    Jacobson SG, Acland GM, Aguirre GD, Aleman TS, Schwartz SB, Cideciyan AV, Zeiss CJ, Komaromy AM, Kaushal S, Roman AJ, Windsor EA, Sumaroka A, Pearce-Kelling SE, Conlon TJ, Chiodo VA, Boye SL, Flotte TR, Maguire AM, Bennett J, Hauswirth WW.
    Mol Ther; 2006 Jun 28; 13(6):1074-84. PubMed ID: 16644289
    [Abstract] [Full Text] [Related]

  • 9. CRB1 heterozygotes with regional retinal dysfunction: implications for genetic testing of leber congenital amaurosis.
    Yzer S, Fishman GA, Racine J, Al-Zuhaibi S, Chakor H, Dorfman A, Szlyk J, Lachapelle P, van den Born LI, Allikmets R, Lopez I, Cremers FP, Koenekoop RK.
    Invest Ophthalmol Vis Sci; 2006 Sep 28; 47(9):3736-44. PubMed ID: 16936081
    [Abstract] [Full Text] [Related]

  • 10. Early-onset severe rod-cone dystrophy in young children with RPE65 mutations.
    Lorenz B, Gyürüs P, Preising M, Bremser D, Gu S, Andrassi M, Gerth C, Gal A.
    Invest Ophthalmol Vis Sci; 2000 Aug 28; 41(9):2735-42. PubMed ID: 10937591
    [Abstract] [Full Text] [Related]

  • 11. Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis.
    Jacobson SG, Boye SL, Aleman TS, Conlon TJ, Zeiss CJ, Roman AJ, Cideciyan AV, Schwartz SB, Komaromy AM, Doobrajh M, Cheung AY, Sumaroka A, Pearce-Kelling SE, Aguirre GD, Kaushal S, Maguire AM, Flotte TR, Hauswirth WW.
    Hum Gene Ther; 2006 Aug 28; 17(8):845-58. PubMed ID: 16942444
    [Abstract] [Full Text] [Related]

  • 12. Clinical phenotypes in carriers of Leber congenital amaurosis mutations.
    Galvin JA, Fishman GA, Stone EM, Koenekoop RK.
    Ophthalmology; 2005 Feb 28; 112(2):349-56. PubMed ID: 15691574
    [Abstract] [Full Text] [Related]

  • 13. Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis.
    Li X, Li W, Dai X, Kong F, Zheng Q, Zhou X, Lü F, Chang B, Rohrer B, Hauswirth WW, Qu J, Pang JJ.
    Invest Ophthalmol Vis Sci; 2011 Jan 28; 52(1):7-15. PubMed ID: 21169527
    [Abstract] [Full Text] [Related]

  • 14. 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 28; 14(4):292-303. PubMed ID: 17024105
    [Abstract] [Full Text] [Related]

  • 15. Self-complementary 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, Umino Y, Rohrer B, Alexander J, Li J, Dai X, Li Q, Chang B, Barlow R, Hauswirth WW.
    Gene Ther; 2010 Jul 28; 17(7):815-26. PubMed ID: 20237510
    [Abstract] [Full Text] [Related]

  • 16. Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.
    Bennicelli J, Wright JF, Komaromy A, Jacobs JB, Hauck B, Zelenaia O, Mingozzi F, Hui D, Chung D, Rex TS, Wei Z, Qu G, Zhou S, Zeiss C, Arruda VR, Acland GM, Dell'Osso LF, High KA, Maguire AM, Bennett J.
    Mol Ther; 2008 Mar 28; 16(3):458-65. PubMed ID: 18209734
    [Abstract] [Full Text] [Related]

  • 17. New views on RPE65 deficiency: the rod system is the source of vision in a mouse model of Leber congenital amaurosis.
    Seeliger MW, Grimm C, Ståhlberg F, Friedburg C, Jaissle G, Zrenner E, Guo H, Remé CE, Humphries P, Hofmann F, Biel M, Fariss RN, Redmond TM, Wenzel A.
    Nat Genet; 2001 Sep 28; 29(1):70-4. PubMed ID: 11528395
    [Abstract] [Full Text] [Related]

  • 18. Successful RPE65 gene replacement and improved visual function in humans.
    Koenekoop RK.
    Ophthalmic Genet; 2008 Sep 28; 29(3):89-91. PubMed ID: 18766986
    [Abstract] [Full Text] [Related]

  • 19. Cortical visual function in the rd12 mouse model of Leber Congenital Amarousis (LCA) after gene replacement therapy to restore retinal function.
    Nusinowitz S, Ridder WH, Pang JJ, Chang B, Noorwez SM, Kaushal S, Hauswirth WW, Heckenlively JR.
    Vision Res; 2006 Oct 28; 46(22):3926-34. PubMed ID: 16814838
    [Abstract] [Full Text] [Related]

  • 20. Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation.
    Aguirre GK, Komáromy AM, Cideciyan AV, Brainard DH, Aleman TS, Roman AJ, Avants BB, Gee JC, Korczykowski M, Hauswirth WW, Acland GM, Aguirre GD, Jacobson SG.
    PLoS Med; 2007 Jun 28; 4(6):e230. PubMed ID: 17594175
    [Abstract] [Full Text] [Related]

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