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Journal Abstract Search

207 related items for PubMed ID: 35001204

  • 1. 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
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  • 4. Short term morphological rescue of the fovea after gene therapy with voretigene neparvovec.
    Kortüm FC, Kempf M, Jung R, Kohl S, Ott S, Kortuem C, Sting K, Stingl K.
    Acta Ophthalmol; 2022 May; 100(3):e807-e812. PubMed ID: 34289237
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  • 5. 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
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  • 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
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  • 10. 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 25; 384(9953):1513-20. PubMed ID: 25030840
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  • 12. 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 25; 120(6):1283-91. PubMed ID: 23474247
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  • 15. Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy.
    Kang C, Scott LJ.
    Mol Diagn Ther; 2020 Aug 25; 24(4):487-495. PubMed ID: 32535767
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  • 17. 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 25; 126(9):1273-1285. PubMed ID: 31443789
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  • 20. 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 25; 63(7):701-705. PubMed ID: 35748082
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