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6. Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis. Pang JJ; Chang B; Kumar A; Nusinowitz S; Noorwez SM; Li J; Rani A; Foster TC; Chiodo VA; Doyle T; Li H; Malhotra R; Teusner JT; McDowell JH; Min SH; Li Q; Kaushal S; Hauswirth WW Mol Ther; 2006 Mar; 13(3):565-72. PubMed ID: 16223604 [TBL] [Abstract][Full Text] [Related]
7. The consortium project to treat RPE65 deficiency in humans. Hauswirth WW Retina; 2005 Dec; 25(8 Suppl):S60. PubMed ID: 16374340 [No Abstract] [Full Text] [Related]
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11. 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. PubMed ID: 15106616 [TBL] [Abstract][Full Text] [Related]
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15. Abnormal retinal architecture in a 33-week-old fetus with LCA and a homozygous C330Y mutation in RPE65. Koenekoop RK Ophthalmic Genet; 2003 Jun; 24(2):125-6. PubMed ID: 12789577 [No Abstract] [Full Text] [Related]
17. 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. PubMed ID: 22183331 [No Abstract] [Full Text] [Related]
19. Impacts of two point mutations of RPE65 from Leber's congenital amaurosis on the stability, subcellular localization and isomerohydrolase activity of RPE65. Chen Y; Moiseyev G; Takahashi Y; Ma JX FEBS Lett; 2006 Jul; 580(17):4200-4. PubMed ID: 16828753 [TBL] [Abstract][Full Text] [Related]
20. 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. PubMed ID: 21299439 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]