These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
340 related articles for article (PubMed ID: 29869534)
1. Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy. Pennesi ME; Weleber RG; Yang P; Whitebirch C; Thean B; Flotte TR; Humphries M; Chegarnov E; Beasley KN; Stout JT; Chulay JD Hum Gene Ther; 2018 Dec; 29(12):1428-1437. PubMed ID: 29869534 [TBL] [Abstract][Full Text] [Related]
2. 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. PubMed ID: 27102010 [TBL] [Abstract][Full Text] [Related]
3. Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Jacobson SG; Cideciyan AV; Ratnakaram R; Heon E; Schwartz SB; Roman AJ; Peden MC; Aleman TS; Boye SL; Sumaroka A; Conlon TJ; Calcedo R; Pang JJ; Erger KE; Olivares MB; Mullins CL; Swider M; Kaushal S; Feuer WJ; Iannaccone A; Fishman GA; Stone EM; Byrne BJ; Hauswirth WW Arch Ophthalmol; 2012 Jan; 130(1):9-24. PubMed ID: 21911650 [TBL] [Abstract][Full Text] [Related]
4. Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2. Testa F; Maguire AM; Rossi S; Pierce EA; Melillo P; Marshall K; Banfi S; Surace EM; Sun J; Acerra C; Wright JF; Wellman J; High KA; Auricchio A; Bennett J; Simonelli F Ophthalmology; 2013 Jun; 120(6):1283-91. PubMed ID: 23474247 [TBL] [Abstract][Full Text] [Related]
5. Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis. Le Meur G; Lebranchu P; Billaud F; Adjali O; Schmitt S; Bézieau S; Péréon Y; Valabregue R; Ivan C; Darmon C; Moullier P; Rolling F; Weber M Mol Ther; 2018 Jan; 26(1):256-268. PubMed ID: 29033008 [TBL] [Abstract][Full Text] [Related]
6. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hauswirth WW; Aleman TS; Kaushal S; Cideciyan AV; Schwartz SB; Wang L; Conlon TJ; Boye SL; Flotte TR; Byrne BJ; Jacobson SG Hum Gene Ther; 2008 Oct; 19(10):979-90. PubMed ID: 18774912 [TBL] [Abstract][Full Text] [Related]
7. Evaluation of Ocular Gene Therapy in an Italian Patient Affected by Congenital Leber Amaurosis Type 2 Treated in Both Eyes. Testa F; Maguire AM; Rossi S; Marshall K; Auricchio A; Melillo P; Bennett J; Simonelli F Adv Exp Med Biol; 2016; 854():533-9. PubMed ID: 26427456 [TBL] [Abstract][Full Text] [Related]
8. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Bennett J; Wellman J; Marshall KA; McCague S; Ashtari M; DiStefano-Pappas J; Elci OU; Chung DC; Sun J; Wright JF; Cross DR; Aravand P; Cyckowski LL; Bennicelli JL; Mingozzi F; Auricchio A; Pierce EA; Ruggiero J; Leroy BP; Simonelli F; High KA; Maguire AM Lancet; 2016 Aug; 388(10045):661-72. PubMed ID: 27375040 [TBL] [Abstract][Full Text] [Related]
9. A Cross-Sectional and Longitudinal Study of Retinal Sensitivity in RPE65-Associated Leber Congenital Amaurosis. Kumaran N; Rubin GS; Kalitzeos A; Fujinami K; Bainbridge JWB; Weleber RG; Michaelides M Invest Ophthalmol Vis Sci; 2018 Jul; 59(8):3330-3339. PubMed ID: 30025081 [TBL] [Abstract][Full Text] [Related]
10. Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials. Maguire AM; Russell S; Wellman JA; Chung DC; Yu ZF; Tillman A; Wittes J; Pappas J; Elci O; Marshall KA; McCague S; Reichert H; Davis M; Simonelli F; Leroy BP; Wright JF; High KA; Bennett J Ophthalmology; 2019 Sep; 126(9):1273-1285. PubMed ID: 31443789 [TBL] [Abstract][Full Text] [Related]
11. 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; 372(20):1887-97. PubMed ID: 25938638 [TBL] [Abstract][Full Text] [Related]
12. The Status of RPE65 Gene Therapy Trials: Safety and Efficacy. Pierce EA; Bennett J Cold Spring Harb Perspect Med; 2015 Jan; 5(9):a017285. PubMed ID: 25635059 [TBL] [Abstract][Full Text] [Related]
13. 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; 110(6):E517-25. PubMed ID: 23341635 [TBL] [Abstract][Full Text] [Related]
14. The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients. Ashtari M; Nikonova ES; Marshall KA; Young GJ; Aravand P; Pan W; Ying GS; Willett AE; Mahmoudian M; Maguire AM; Bennett J Ophthalmology; 2017 Jun; 124(6):873-883. PubMed ID: 28237426 [TBL] [Abstract][Full Text] [Related]
15. 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. PubMed ID: 24028205 [TBL] [Abstract][Full Text] [Related]
16. Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial. Koenekoop RK; Sui R; Sallum J; van den Born LI; Ajlan R; Khan A; den Hollander AI; Cremers FP; Mendola JD; Bittner AK; Dagnelie G; Schuchard RA; Saperstein DA Lancet; 2014 Oct; 384(9953):1513-20. PubMed ID: 25030840 [TBL] [Abstract][Full Text] [Related]
17. 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. PubMed ID: 19583479 [TBL] [Abstract][Full Text] [Related]
18. 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. PubMed ID: 27653967 [TBL] [Abstract][Full Text] [Related]
19. Effect of gene therapy on visual function in Leber's congenital amaurosis. Bainbridge JW; Smith AJ; Barker SS; Robbie S; Henderson R; Balaggan K; Viswanathan A; Holder GE; Stockman A; Tyler N; Petersen-Jones S; Bhattacharya SS; Thrasher AJ; Fitzke FW; Carter BJ; Rubin GS; Moore AT; Ali RR N Engl J Med; 2008 May; 358(21):2231-9. PubMed ID: 18441371 [TBL] [Abstract][Full Text] [Related]
20. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Simonelli F; Maguire AM; Testa F; Pierce EA; Mingozzi F; Bennicelli JL; Rossi S; Marshall K; Banfi S; Surace EM; Sun J; Redmond TM; Zhu X; Shindler KS; Ying GS; Ziviello C; Acerra C; Wright JF; McDonnell JW; High KA; Bennett J; Auricchio A Mol Ther; 2010 Mar; 18(3):643-50. PubMed ID: 19953081 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]