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Journal Abstract Search
204 related items for PubMed ID: 35748082
1. Short-Term Outcomes of the First in Vivo Gene Therapy for RPE65-Mediated Retinitis Pigmentosa. Kwak JJ, Kim HR, Byeon SH. Yonsei Med J; 2022 Jul; 63(7):701-705. PubMed ID: 35748082 [Abstract] [Full Text] [Related]
2. Perifoveal Chorioretinal Atrophy after Subretinal Voretigene Neparvovec-rzyl for RPE65-Mediated Leber Congenital Amaurosis. Gange WS, Sisk RA, Besirli CG, Lee TC, Havunjian M, Schwartz H, Borchert M, Sengillo JD, Mendoza C, Berrocal AM, Nagiel A. Ophthalmol Retina; 2022 Jan; 6(1):58-64. PubMed ID: 33838313 [Abstract] [Full Text] [Related]
3. The first gene therapy for RPE65 biallelic dystrophy with voretigene neparvovec-rzyl in Brazil. Ferraz Sallum JM, Godoy J, Kondo A, Kutner JM, Vasconcelos H, Maia A. Ophthalmic Genet; 2022 Aug; 43(4):550-554. PubMed ID: 35416119 [Abstract] [Full Text] [Related]
4. RPE65 mutations in Leber congenital amaurosis, early-onset severe retinal dystrophy, and retinitis pigmentosa from a tertiary eye care center in India. Parameswarappa DC, Bagga DK, Upadhyaya A, Balasubramanian J, Pochaboina V, Muthineni V, Jalali S, Kannabiran C. Ophthalmic Genet; 2024 Jun; 45(3):303-312. PubMed ID: 38323530 [Abstract] [Full Text] [Related]
6. 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 [Abstract] [Full Text] [Related]
7. 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 01; 137(12):1381-1388. PubMed ID: 31580392 [Abstract] [Full Text] [Related]
8. Current Management of Patients with RPE65 Mutation-Associated Inherited Retinal Degenerations in Europe: Results of a Multinational Survey by the European Vision Institute Clinical Research Network. Lorenz B, Tavares J, van den Born LI, Marques JP, Scholl HPN, EVICR.net Group. Ophthalmic Res; 2021 Dec 01; 64(5):740-753. PubMed ID: 33684911 [Abstract] [Full Text] [Related]
10. Subretinal Injection of Voretigene Neparvovec-rzyl in a Patient With RPE65-Associated Leber's Congenital Amaurosis. Hussain RM, Tran KD, Maguire AM, Berrocal AM. Ophthalmic Surg Lasers Imaging Retina; 2019 Oct 01; 50(10):661-663. PubMed ID: 31671202 [Abstract] [Full Text] [Related]
11. Properties and Therapeutic Implications of an Enigmatic D477G RPE65 Variant Associated with Autosomal Dominant Retinitis Pigmentosa. Kiang AS, Kenna PF, Humphries MM, Ozaki E, Koenekoop RK, Campbell M, Farrar GJ, Humphries P. Genes (Basel); 2020 Nov 27; 11(12):. PubMed ID: 33261050 [Abstract] [Full Text] [Related]
12. Surgical Observations From the First 120 Cases of Subretinal Gene Therapy for Inherited Retinal Diseases. Gregori NZ, Davis JL. Retina; 2023 Sep 01; 43(9):1608-1611. PubMed ID: 33394965 [Abstract] [Full Text] [Related]
13. Voretigene neparvovec-rzyl for treatment of RPE65-mediated inherited retinal diseases: a model for ocular gene therapy development. Ciulla TA, Hussain RM, Berrocal AM, Nagiel A. Expert Opin Biol Ther; 2020 Jun 01; 20(6):565-578. PubMed ID: 32149547 [Abstract] [Full Text] [Related]
14. Chorioretinal atrophy following voretigene neparvovec despite the presence of fundus autofluorescence. Kolesnikova M, Lima de Carvalho JR, Parmann R, Kim AH, Mahajan VB, Tsang SH, Sparrow JR. Mol Genet Genomic Med; 2022 Nov 01; 10(11):e2038. PubMed ID: 36225124 [Abstract] [Full Text] [Related]
15. [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 Nov 01; 153(2):276-278. PubMed ID: 28474714 [Abstract] [Full Text] [Related]
19. 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 01; 130(1):9-24. PubMed ID: 21911650 [Abstract] [Full Text] [Related]