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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. Bainbridge JW, et al. Among authors: mowat fm. N Engl J Med. 2015 May 14;372(20):1887-97. doi: 10.1056/NEJMoa1414221. Epub 2015 May 4. N Engl J Med. 2015. PMID: 25938638 Free PMC article. Clinical Trial.
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. Mowat FM, et al. Gene Ther. 2013 May;20(5):545-55. doi: 10.1038/gt.2012.63. Epub 2012 Sep 6. Gene Ther. 2013. PMID: 22951453
46 results