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Items: 1 to 20 of 142

1.

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.

2.

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
3.

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.

Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JI, Hauck B, Zelenaia O, Zhu X, Raffini L, Coppieters F, De Baere E, Shindler KS, Volpe NJ, Surace EM, Acerra C, Lyubarsky A, Redmond TM, Stone E, Sun J, McDonnell JW, Leroy BP, Simonelli F, Bennett J.

Lancet. 2009 Nov 7;374(9701):1597-605. doi: 10.1016/S0140-6736(09)61836-5. Epub 2009 Oct 23. Erratum in: Lancet. 2010 Jan 2;375(9708):30.

4.

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.

5.

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.

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.

7.

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. doi: 10.1089/hum.2008.107.

8.

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
9.

In utero gene therapy rescues vision in a murine model of congenital blindness.

Dejneka NS, Surace EM, Aleman TS, Cideciyan AV, Lyubarsky A, Savchenko A, Redmond TM, Tang W, Wei Z, Rex TS, Glover E, Maguire AM, Pugh EN Jr, Jacobson SG, Bennett J.

Mol Ther. 2004 Feb;9(2):182-8.

10.

Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness.

Amado D, Mingozzi F, Hui D, Bennicelli JL, Wei Z, Chen Y, Bote E, Grant RL, Golden JA, Narfstrom K, Syed NA, Orlin SE, High KA, Maguire AM, Bennett J.

Sci Transl Med. 2010 Mar 3;2(21):21ra16. doi: 10.1126/scitranslmed.3000659.

11.

Using the NAFX to measure the effectiveness over time of gene therapy in canine LCA.

Jacobs JB, Dell'Osso LF, Wang ZI, Acland GM, Bennett J.

Invest Ophthalmol Vis Sci. 2009 Oct;50(10):4685-92. doi: 10.1167/iovs.09-3387. Epub 2009 May 20.

12.

Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice.

Barker SE, Broderick CA, Robbie SJ, Duran Y, Natkunarajah M, Buch P, Balaggan KS, MacLaren RE, Bainbridge JW, Smith AJ, Ali RR.

J Gene Med. 2009 Jun;11(6):486-97. doi: 10.1002/jgm.1327.

13.

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
14.

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.

15.

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
16.

AAV2 gene therapy readministration in three adults with congenital blindness.

Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, McCague S, Pierce EA, Chen Y, Bennicelli JL, Zhu X, Ying GS, Sun J, Wright JF, Auricchio A, Simonelli F, Shindler KS, Mingozzi F, High KA, Maguire AM.

Sci Transl Med. 2012 Feb 8;4(120):120ra15. doi: 10.1126/scitranslmed.3002865.

17.

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;4(6):e230.

18.

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.

19.

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.

20.

Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65.

Georgiadis A, Duran Y, Ribeiro J, Abelleira-Hervas L, Robbie SJ, Sünkel-Laing B, Fourali S, Gonzalez-Cordero A, Cristante E, Michaelides M, Bainbridge JW, Smith AJ, Ali RR.

Gene Ther. 2016 Dec;23(12):857-862. doi: 10.1038/gt.2016.66. Epub 2016 Sep 22. Erratum in: Gene Ther. 2018 Sep;25(6):450.

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