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Items: 1 to 20 of 26

1.

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. Erratum in: Lancet. 2010 Jan 2;375(9708):30.

2.

Gene therapy: light is finally in the tunnel.

Cao H, Molday RS, Hu J.

Protein Cell. 2011 Dec;2(12):973-89. doi: 10.1007/s13238-011-1126-y. Review.

3.

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. Review.

4.

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
5.

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.

6.

Gene therapy for Leber congenital amaurosis.

Bennett J.

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

PMID:
14750605
7.

[Past, present, and future in Leber's hereditary optic neuropathy].

Oguchi Y.

Nippon Ganka Gakkai Zasshi. 2001 Dec;105(12):809-27. Review. Japanese.

PMID:
11802455
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.

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
10.

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
11.

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. Review.

PMID:
22644094
12.

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
13.

[From gene to disease; Leber congenital amaurosis (LCA)].

Yzer S, van den Born LI, Cremers FP, den Hollander AI.

Ned Tijdschr Geneeskd. 2005 Oct 15;149(42):2334-7. Review. Dutch.

PMID:
16261712
14.

Leber's congenital amaurosis: an update.

Fazzi E, Signorini SG, Scelsa B, Bova SM, Lanzi G.

Eur J Paediatr Neurol. 2003;7(1):13-22. Review.

PMID:
12615170
15.

Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients.

Colella P, Auricchio A.

Hum Gene Ther. 2012 Aug;23(8):796-807. doi: 10.1089/hum.2012.123. Review.

16.

Follow-up and diagnostic reappraisal of 75 patients with Leber's congenital amaurosis.

Lambert SR, Kriss A, Taylor D, Coffey R, Pembrey M.

Am J Ophthalmol. 1989 Jun 15;107(6):624-31. Review.

PMID:
2658617
17.

Gene therapy for the eye focus on mutation-independent approaches.

Dalkara D, Duebel J, Sahel JA.

Curr Opin Neurol. 2015 Feb;28(1):51-60. doi: 10.1097/WCO.0000000000000168. Review.

PMID:
25545056
18.

Let There Be Light: Gene and Cell Therapy for Blindness.

Dalkara D, Goureau O, Marazova K, Sahel JA.

Hum Gene Ther. 2016 Feb;27(2):134-47. doi: 10.1089/hum.2015.147. Review.

PMID:
26751519
19.

[Gene therapy as a treatment concept for inherited retinal diseases].

Bellingrath JS, Fischer MD.

Ophthalmologe. 2015 Sep;112(9):720-7. doi: 10.1007/s00347-015-0121-8. Review. German.

PMID:
26293194
20.

Dog models for blinding inherited retinal dystrophies.

Petersen-Jones SM, Komáromy AM.

Hum Gene Ther Clin Dev. 2015 Mar;26(1):15-26. doi: 10.1089/humc.2014.155. Review.

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