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Items: 1 to 20 of 30

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.

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

Hufnagel RB, Ahmed ZM, CorrĂȘa 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
3.

Molecular genetics of Leber congenital amaurosis.

Cremers FP, van den Hurk JA, den Hollander AI.

Hum Mol Genet. 2002 May 15;11(10):1169-76. Review.

4.

Gene therapy for Leber congenital amaurosis.

Bennett J.

Novartis Found Symp. 2004;255:195-202; discussion 202-7. Review.

PMID:
14750605
5.

Leber congenital amaurosis: genes, proteins and disease mechanisms.

den Hollander AI, Roepman R, Koenekoop RK, Cremers FP.

Prog Retin Eye Res. 2008 Jul;27(4):391-419. doi: 10.1016/j.preteyeres.2008.05.003. Epub 2008 Jun 1. Review.

PMID:
18632300
6.

An overview of Leber congenital amaurosis: a model to understand human retinal development.

Koenekoop RK.

Surv Ophthalmol. 2004 Jul-Aug;49(4):379-98. Review.

PMID:
15231395
7.

RPE65: role in the visual cycle, human retinal disease, and gene therapy.

Cai X, Conley SM, Naash MI.

Ophthalmic Genet. 2009 Jun;30(2):57-62. doi: 10.1080/13816810802626399. Review.

8.

Recent advances in early-onset severe retinal degeneration: more than just basic research.

Preising MN, Heegaard S.

Trends Mol Med. 2004 Feb;10(2):51-4. Review.

PMID:
15106616
9.

Leber congenital amaurosis.

Perrault I, Rozet JM, Gerber S, Ghazi I, Leowski C, Ducroq D, Souied E, Dufier JL, Munnich A, Kaplan J.

Mol Genet Metab. 1999 Oct;68(2):200-8. Review.

PMID:
10527670
10.

Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy.

Cideciyan AV.

Prog Retin Eye Res. 2010 Sep;29(5):398-427. doi: 10.1016/j.preteyeres.2010.04.002. Epub 2010 Apr 24. Review.

11.

Gene therapy for Leber congenital amaurosis.

Dejneka NS, Surace EM, Bennett J.

Adv Exp Med Biol. 2003;533:415-22. Review. No abstract available.

PMID:
15180293
12.

Gene therapy for vision loss -- recent developments.

Stieger K, Lorenz B.

Discov Med. 2010 Nov;10(54):425-33. Review.

13.

Clinical gene therapy for the treatment of RPE65-associated Leber congenital amaurosis.

Stein L, Roy K, Lei L, Kaushal S.

Expert Opin Biol Ther. 2011 Mar;11(3):429-39. doi: 10.1517/14712598.2011.557358. Review.

PMID:
21299439
14.

Naturally occurring animal models with outer retina phenotypes.

Baehr W, Frederick JM.

Vision Res. 2009 Nov;49(22):2636-52. doi: 10.1016/j.visres.2009.04.008. Epub 2009 Apr 16. Review.

15.

Manufacturing and regulatory strategies for clinical AAV2-hRPE65.

Wright JF, Wellman J, High KA.

Curr Gene Ther. 2010 Oct;10(5):341-9. Review.

PMID:
20712582
16.

[Animal models of human retinal and optic nerve diseases analysed using electroretinography].

Kondo M.

Nippon Ganka Gakkai Zasshi. 2010 Mar;114(3):248-78, discussion 279. Review. Japanese.

PMID:
20387538
17.

Leber congenital amaurosis: clinical correlations with genotypes, gene therapy trials update, and future directions.

Chung DC, Traboulsi EI.

J AAPOS. 2009 Dec;13(6):587-92. doi: 10.1016/j.jaapos.2009.10.004. Review.

PMID:
20006823
18.

Gene augmentation trials using the Rpe65-deficient dog: contributions towards development and refinement of human clinical trials.

Petersen-Jones SM, Annear MJ, Bartoe JT, Mowat FM, Barker SE, Smith AJ, Bainbridge JW, Ali RR.

Adv Exp Med Biol. 2012;723:177-82. doi: 10.1007/978-1-4614-0631-0_24. Review. No abstract available.

PMID:
22183331
19.

An analysis of retinal gene therapy clinical trials.

MacLaren RE.

Curr Opin Mol Ther. 2009 Oct;11(5):540-6. Review.

20.

The Status of RPE65 Gene Therapy Trials: Safety and Efficacy.

Pierce EA, Bennett J.

Cold Spring Harb Perspect Med. 2015 Jan 29;5(9):a017285. doi: 10.1101/cshperspect.a017285. Review.

PMID:
25635059
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