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Items: 18

1.

Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year.

Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, Windsor EA, Conlon TJ, Sumaroka A, Pang JJ, Roman AJ, Byrne BJ, Jacobson SG.

Hum Gene Ther. 2009 Sep;20(9):999-1004. doi: 10.1089/hum.2009.086.

2.

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
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. Epub 2010 Apr 24. Review.

4.

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
5.

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
6.

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. Epub 2012 Jan 10. Review.

7.

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.

8.

Available Evidence on Leber Congenital Amaurosis and Gene Therapy.

Alkharashi M, Fulton AB.

Semin Ophthalmol. 2017;32(1):14-21. doi: 10.1080/08820538.2016.1228383. Epub 2016 Sep 29. Review.

PMID:
27686653
9.

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
10.

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
11.

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.

12.

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.

13.

Cone-rod dystrophy caused by a novel homozygous RPE65 mutation in Leber congenital amaurosis.

Jakobsson C, Othman IS, Munier FL, Schorderet DF, Abouzeid H.

Klin Monbl Augenheilkd. 2014 Apr;231(4):405-10. doi: 10.1055/s-0034-1368221. Epub 2014 Apr 25. Review.

PMID:
24771178
14.

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.

15.

Membrane-binding and enzymatic properties of RPE65.

Kiser PD, Palczewski K.

Prog Retin Eye Res. 2010 Sep;29(5):428-42. doi: 10.1016/j.preteyeres.2010.03.002. Epub 2010 Mar 19. Review.

16.

Gene supplementation therapy for recessive forms of inherited retinal dystrophies.

Smith AJ, Bainbridge JW, Ali RR.

Gene Ther. 2012 Feb;19(2):154-61. doi: 10.1038/gt.2011.161. Epub 2011 Oct 27. Review.

PMID:
22033465
17.

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. Epub 2015 Feb 11. Review.

18.

A comprehensive review of retinal gene therapy.

Boye SE, Boye SL, Lewin AS, Hauswirth WW.

Mol Ther. 2013 Mar;21(3):509-19. doi: 10.1038/mt.2012.280. Epub 2013 Jan 29. Review. Erratum in: Mol Ther. 2013 Nov;21(11):2130.

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