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Items: 1 to 20 of 244

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.

PMID:
17960108
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.

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;51(10):5304-13. doi: 10.1167/iovs.10-5559. Epub 2010 May 19.

PMID:
20484585
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;13(3):565-72. Epub 2005 Oct 11.

5.

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.

6.

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
7.

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
8.

Impairment of the transient pupillary light reflex in Rpe65(-/-) mice and humans with leber congenital amaurosis.

Aleman TS, Jacobson SG, Chico JD, Scott ML, Cheung AY, Windsor EA, Furushima M, Redmond TM, Bennett J, Palczewski K, Cideciyan AV.

Invest Ophthalmol Vis Sci. 2004 Apr;45(4):1259-71.

PMID:
15037595
9.

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
10.

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 5;52(1):7-15. doi: 10.1167/iovs.10-6138. Print 2011 Jan.

11.

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;13(6):1074-84. Epub 2006 Apr 27.

12.

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;47(9):3736-44.

PMID:
16936081
13.

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;41(9):2735-42.

PMID:
10937591
14.

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;17(8):845-58.

PMID:
16942444
15.

Clinical phenotypes in carriers of Leber congenital amaurosis mutations.

Galvin JA, Fishman GA, Stone EM, Koenekoop RK.

Ophthalmology. 2005 Feb;112(2):349-56.

PMID:
15691574
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;16(3):458-65. doi: 10.1038/sj.mt.6300389. Epub 2008 Jan 22.

17.

Defining the residual vision in leber congenital amaurosis caused by RPE65 mutations.

Jacobson SG, Aleman TS, Cideciyan AV, Roman AJ, Sumaroka A, Windsor EA, Schwartz SB, Heon E, Stone EM.

Invest Ophthalmol Vis Sci. 2009 May;50(5):2368-75. doi: 10.1167/iovs.08-2696. Epub 2008 Dec 30.

18.

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
19.

Retinal degeneration in Aipl1-deficient mice: a new genetic model of Leber congenital amaurosis.

Dyer MA, Donovan SL, Zhang J, Gray J, Ortiz A, Tenney R, Kong J, Allikmets R, Sohocki MM.

Brain Res Mol Brain Res. 2004 Dec 20;132(2):208-20.

PMID:
15582159
20.

Gene therapy for Leber congenital amaurosis: advances and future directions.

Hufnagel RB, Ahmed ZM, Corrêa ZM, Sisk RA.

Graefes Arch Clin Exp Ophthalmol. 2012 Aug;250(8):1117-28. doi: 10.1007/s00417-012-2028-2. Epub 2012 May 29. Review.

PMID:
22644094

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