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Items: 1 to 20 of 70

1.

Reply to Townes-Anderson: RPE65 gene therapy does not alter the natural history of retinal degeneration.

Cideciyan AV, Jacobson SG, Beltran WA, Hauswirth WW, Aguirre GD.

Proc Natl Acad Sci U S A. 2013 May 7;110(19):E1706. No abstract available.

2.

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement.

Cideciyan AV, Jacobson SG, Beltran WA, Sumaroka A, Swider M, Iwabe S, Roman AJ, Olivares MB, Schwartz SB, Komáromy AM, Hauswirth WW, Aguirre GD.

Proc Natl Acad Sci U S A. 2013 Feb 5;110(6):E517-25. doi: 10.1073/pnas.1218933110. Epub 2013 Jan 22.

3.

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
4.

Increased levels of gene therapy may not be beneficial in retinal disease.

Townes-Anderson E.

Proc Natl Acad Sci U S A. 2013 May 7;110(19):E1705. doi: 10.1073/pnas.1303746110. Epub 2013 Apr 3. No abstract available.

5.
6.

Gene therapy for eye as regenerative medicine? Lessons from RPE65 gene therapy for Leber's Congenital Amaurosis.

Rakoczy EP, Narfström K.

Int J Biochem Cell Biol. 2014 Nov;56:153-7. doi: 10.1016/j.biocel.2014.09.022. Epub 2014 Oct 5.

PMID:
25286304
7.

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
8.

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.

9.

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.

10.

Investor Outlook: Focus on Upcoming LCA2 Gene Therapy Phase III Results.

Schimmer J, Breazzano S.

Hum Gene Ther Clin Dev. 2015 Sep;26(3):144-9. doi: 10.1089/humc.2015.29001.sch.

PMID:
26390089
11.

Pseudo-fovea formation after gene therapy for RPE65-LCA.

Cideciyan AV, Aguirre GK, Jacobson SG, Butt OH, Schwartz SB, Swider M, Roman AJ, Sadigh S, Hauswirth WW.

Invest Ophthalmol Vis Sci. 2014 Dec 23;56(1):526-37. doi: 10.1167/iovs.14-15895.

12.

RPE65 gene therapy slows cone loss in Rpe65-deficient dogs.

Mowat FM, Breuwer AR, Bartoe JT, Annear MJ, Zhang Z, Smith AJ, Bainbridge JW, Petersen-Jones SM, Ali RR.

Gene Ther. 2013 May;20(5):545-55. doi: 10.1038/gt.2012.63. Epub 2012 Sep 6.

PMID:
22951453
13.

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.

14.

Long-term effect of gene therapy on Leber's congenital amaurosis.

Bainbridge JW, Mehat MS, Sundaram V, Robbie SJ, Barker SE, Ripamonti C, Georgiadis A, Mowat FM, Beattie SG, Gardner PJ, Feathers KL, Luong VA, Yzer S, Balaggan K, Viswanathan A, de Ravel TJ, Casteels I, Holder GE, Tyler N, Fitzke FW, Weleber RG, Nardini M, Moore AT, Thompson DA, Petersen-Jones SM, Michaelides M, van den Born LI, Stockman A, Smith AJ, Rubin G, Ali RR.

N Engl J Med. 2015 May 14;372(20):1887-97. doi: 10.1056/NEJMoa1414221. Epub 2015 May 4.

15.

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.

16.

Investor Outlook: Significance of the Positive LCA2 Gene Therapy Phase III Results.

Schimmer J, Breazzano S.

Hum Gene Ther Clin Dev. 2015 Dec;26(4):208-10. doi: 10.1089/humc.2015.29004.sch.

PMID:
26684444
17.

Long-term effects of retinal gene therapy in childhood blindness.

Wright AF.

N Engl J Med. 2015 May 14;372(20):1954-5. doi: 10.1056/NEJMe1503419. Epub 2015 May 4. No abstract available.

PMID:
25938534
18.

Successful gene therapy in older Rpe65-deficient dogs following subretinal injection of an adeno-associated vector expressing RPE65.

Annear MJ, Mowat FM, Bartoe JT, Querubin J, Azam SA, Basche M, Curran PG, Smith AJ, Bainbridge JW, Ali RR, Petersen-Jones SM.

Hum Gene Ther. 2013 Oct;24(10):883-93. doi: 10.1089/hum.2013.146.

PMID:
24028205
19.

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
20.

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.

Weleber RG, Pennesi ME, Wilson DJ, Kaushal S, Erker LR, Jensen L, McBride MT, Flotte TR, Humphries M, Calcedo R, Hauswirth WW, Chulay JD, Stout JT.

Ophthalmology. 2016 Jul;123(7):1606-20. doi: 10.1016/j.ophtha.2016.03.003. Epub 2016 Apr 19.

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