835 related articles for article (PubMed ID: 21911650)
21. Molecular anthropology meets genetic medicine to treat blindness in the North African Jewish population: human gene therapy initiated in Israel.
Banin E; Bandah-Rozenfeld D; Obolensky A; Cideciyan AV; Aleman TS; Marks-Ohana D; Sela M; Boye S; Sumaroka A; Roman AJ; Schwartz SB; Hauswirth WW; Jacobson SG; Hemo I; Sharon D
Hum Gene Ther; 2010 Dec; 21(12):1749-57. PubMed ID: 20604683
[TBL] [Abstract][Full Text] [Related]
22. 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]
23. Retinal disease in Rpe65-deficient mice: comparison to human leber congenital amaurosis due to RPE65 mutations.
Caruso RC; Aleman TS; Cideciyan AV; Roman AJ; Sumaroka A; Mullins CL; Boye SL; Hauswirth WW; Jacobson SG
Invest Ophthalmol Vis Sci; 2010 Oct; 51(10):5304-13. PubMed ID: 20484585
[TBL] [Abstract][Full Text] [Related]
24. Human retinal disease from AIPL1 gene mutations: foveal cone loss with minimal macular photoreceptors and rod function remaining.
Jacobson SG; Cideciyan AV; Aleman TS; Sumaroka A; Roman AJ; Swider M; Schwartz SB; Banin E; Stone EM
Invest Ophthalmol Vis Sci; 2011 Jan; 52(1):70-9. PubMed ID: 20702822
[TBL] [Abstract][Full Text] [Related]
25. Photoreceptor layer topography in children with leber congenital amaurosis caused by RPE65 mutations.
Jacobson SG; Cideciyan AV; Aleman TS; Sumaroka A; Windsor EA; Schwartz SB; Heon E; Stone EM
Invest Ophthalmol Vis Sci; 2008 Oct; 49(10):4573-7. PubMed ID: 18539930
[TBL] [Abstract][Full Text] [Related]
26. Gene therapy in the second eye of RPE65-deficient dogs improves retinal function.
Annear MJ; Bartoe JT; Barker SE; Smith AJ; Curran PG; Bainbridge JW; Ali RR; Petersen-Jones SM
Gene Ther; 2011 Jan; 18(1):53-61. PubMed ID: 20703309
[TBL] [Abstract][Full Text] [Related]
27. 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. PubMed ID: 22644094
[TBL] [Abstract][Full Text] [Related]
28. Utility of In Vitro Mutagenesis of RPE65 Protein for Verification of Mutational Pathogenicity Before Gene Therapy.
Yang U; Gentleman S; Gai X; Gorin MB; Borchert MS; Lee TC; Villanueva A; Koenekoop R; Maguire AM; Bennett J; Redmond TM; Nagiel A
JAMA Ophthalmol; 2019 Dec; 137(12):1381-1388. PubMed ID: 31580392
[TBL] [Abstract][Full Text] [Related]
29. Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye.
Li W; Kong F; Li X; Dai X; Liu X; Zheng Q; Wu R; Zhou X; Lü F; Chang B; Li Q; Hauswirth WW; Qu J; Pang JJ
Mol Vis; 2009; 15():267-75. PubMed ID: 19190735
[TBL] [Abstract][Full Text] [Related]
30. 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]
31. 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. PubMed ID: 20399883
[TBL] [Abstract][Full Text] [Related]
32. Overexpression of Type 3 Iodothyronine Deiodinase Reduces Cone Death in the Leber Congenital Amaurosis Model Mice.
Yang F; Ma H; Boye SL; Hauswirth WW; Ding XQ
Adv Exp Med Biol; 2018; 1074():125-131. PubMed ID: 29721936
[TBL] [Abstract][Full Text] [Related]
33. The human visual cortex responds to gene therapy-mediated recovery of retinal function.
Ashtari M; Cyckowski LL; Monroe JF; Marshall KA; Chung DC; Auricchio A; Simonelli F; Leroy BP; Maguire AM; Shindler KS; Bennett J
J Clin Invest; 2011 Jun; 121(6):2160-8. PubMed ID: 21606598
[TBL] [Abstract][Full Text] [Related]
34. Differential macular morphology in patients with RPE65-, CEP290-, GUCY2D-, and AIPL1-related Leber congenital amaurosis.
Pasadhika S; Fishman GA; Stone EM; Lindeman M; Zelkha R; Lopez I; Koenekoop RK; Shahidi M
Invest Ophthalmol Vis Sci; 2010 May; 51(5):2608-14. PubMed ID: 19959640
[TBL] [Abstract][Full Text] [Related]
35. 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; 56(1):526-37. PubMed ID: 25537204
[TBL] [Abstract][Full Text] [Related]
36. Real-world outcomes of voretigene neparvovec treatment in pediatric patients with RPE65-associated Leber congenital amaurosis.
Deng C; Zhao PY; Branham K; Schlegel D; Fahim AT; Jayasundera TK; Khan N; Besirli CG
Graefes Arch Clin Exp Ophthalmol; 2022 May; 260(5):1543-1550. PubMed ID: 35001204
[TBL] [Abstract][Full Text] [Related]
37. 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]
38. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.
Acland GM; Aguirre GD; Bennett J; Aleman TS; Cideciyan AV; Bennicelli J; Dejneka NS; Pearce-Kelling SE; Maguire AM; Palczewski K; Hauswirth WW; Jacobson SG
Mol Ther; 2005 Dec; 12(6):1072-82. PubMed ID: 16226919
[TBL] [Abstract][Full Text] [Related]
39. The phenotype of Severe Early Childhood Onset Retinal Dystrophy (SECORD) from mutation of RPE65 and differentiation from Leber congenital amaurosis.
Weleber RG; Michaelides M; Trzupek KM; Stover NB; Stone EM
Invest Ophthalmol Vis Sci; 2011 Jan; 52(1):292-302. PubMed ID: 20811047
[TBL] [Abstract][Full Text] [Related]
40. 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]
[Previous] [Next] [New Search]