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.
507 related articles for article (PubMed ID: 22644094)
1. 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]
2. An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials. Chiu W; Lin TY; Chang YC; Isahwan-Ahmad Mulyadi Lai H; Lin SC; Ma C; Yarmishyn AA; Lin SC; Chang KJ; Chou YB; Hsu CC; Lin TC; Chen SJ; Chien Y; Yang YP; Hwang DK Int J Mol Sci; 2021 Apr; 22(9):. PubMed ID: 33926102 [TBL] [Abstract][Full Text] [Related]
3. 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]
4. Available Evidence on Leber Congenital Amaurosis and Gene Therapy. Alkharashi M; Fulton AB Semin Ophthalmol; 2017; 32(1):14-21. PubMed ID: 27686653 [TBL] [Abstract][Full Text] [Related]
5. 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]
6. 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]
8. Pharmacological Amelioration of Cone Survival and Vision in a Mouse Model for Leber Congenital Amaurosis. Li S; Samardzija M; Yang Z; Grimm C; Jin M J Neurosci; 2016 May; 36(21):5808-19. PubMed ID: 27225770 [TBL] [Abstract][Full Text] [Related]
9. 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]
10. 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]
11. Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis. Li X; Li W; Dai X; Kong F; Zheng Q; Zhou X; Lü F; Chang B; Rohrer B; Hauswirth WW; Qu J; Pang JJ Invest Ophthalmol Vis Sci; 2011 Jan; 52(1):7-15. PubMed ID: 21169527 [TBL] [Abstract][Full Text] [Related]
12. [Gene therapy for vision restoration in patients with Leber congenital amaurosis (LCA) due to RPE65 gene mutations: beginning the phase IV trial]. Chacón-Camacho ÓF; Zenteno JC Gac Med Mex; 2017; 153(2):276-278. PubMed ID: 28474714 [TBL] [Abstract][Full Text] [Related]
13. 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]
14. 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]
15. 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]
16. 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]
17. 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. PubMed ID: 25286304 [TBL] [Abstract][Full Text] [Related]
18. 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]
19. Leber congenital amaurosis/early-onset severe retinal dystrophy: current management and clinical trials. Daich Varela M; Cabral de Guimaraes TA; Georgiou M; Michaelides M Br J Ophthalmol; 2022 Apr; 106(4):445-451. PubMed ID: 33712480 [TBL] [Abstract][Full Text] [Related]
20. Leber congenital amaurosis/early-onset severe retinal dystrophy: clinical features, molecular genetics and therapeutic interventions. Kumaran N; Moore AT; Weleber RG; Michaelides M Br J Ophthalmol; 2017 Sep; 101(9):1147-1154. PubMed ID: 28689169 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]