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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM. Russell S, et al. Among authors: stone e. Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14. Lancet. 2017. PMID: 28712537 Free PMC article. Clinical Trial.
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.
Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JI, Hauck B, Zelenaia O, Zhu X, Raffini L, Coppieters F, De Baere E, Shindler KS, Volpe NJ, Surace EM, Acerra C, Lyubarsky A, Redmond TM, Stone E, Sun J, McDonnell JW, Leroy BP, Simonelli F, Bennett J. Maguire AM, et al. Among authors: stone e. Lancet. 2009 Nov 7;374(9701):1597-605. doi: 10.1016/S0140-6736(09)61836-5. Epub 2009 Oct 23. Lancet. 2009. PMID: 19854499 Free PMC article. Clinical Trial.
Safety and efficacy of gene transfer for Leber's congenital amaurosis.
Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell'Osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A, High KA, Bennett J. Maguire AM, et al. Among authors: stone e. N Engl J Med. 2008 May 22;358(21):2240-8. doi: 10.1056/NEJMoa0802315. Epub 2008 Apr 27. N Engl J Med. 2008. PMID: 18441370 Free PMC article. Clinical Trial.
Union makes strength: a worldwide collaborative genetic and clinical study to provide a comprehensive survey of RD3 mutations and delineate the associated phenotype.
Perrault I, Estrada-Cuzcano A, Lopez I, Kohl S, Li S, Testa F, Zekveld-Vroon R, Wang X, Pomares E, Andorf J, Aboussair N, Banfi S, Delphin N, den Hollander AI, Edelson C, Florijn R, Jean-Pierre M, Leowski C, Megarbane A, Villanueva C, Flores B, Munnich A, Ren H, Zobor D, Bergen A, Chen R, Cremers FP, Gonzalez-Duarte R, Koenekoop RK, Simonelli F, Stone E, Wissinger B, Zhang Q, Kaplan J, Rozet JM. Perrault I, et al. Among authors: stone e. PLoS One. 2013;8(1):e51622. doi: 10.1371/journal.pone.0051622. Epub 2013 Jan 7. PLoS One. 2013. PMID: 23308101 Free PMC article.
Biofeedback-Based Videogame May Improve Rage Attacks in Tourette Syndrome.
Vermilion J, Walsh N, Tae M, Peechatka A, Kahn J, Ragnio J, Stone E, Mink JW. Vermilion J, et al. Among authors: stone e. J Child Adolesc Psychopharmacol. 2025 Jan 6. doi: 10.1089/cap.2024.0084. Online ahead of print. J Child Adolesc Psychopharmacol. 2025. PMID: 39757878
Reciprocal links between methionine metabolism, DNA repair and therapy resistance in glioblastoma.
Korimerla N, Meghdadi B, Haq I, Wilder-Romans K, Xu J, Becker N, Zhu Z, Kalev P, Qi N, Evans C, Kachman M, Zhao Z, Lin A, Scott AJ, O'Brien A, Kothari A, Sajjakulnukit P, Zhang L, Palavalasa S, Peterson ER, Hyer ML, Marjon K, Sleger T, Morgan MA, Lyssiotis CA, Stone EM, Ferris SP, Lawrence TS, Nagrath D, Zhou W, Wahl DR. Korimerla N, et al. Among authors: stone em. bioRxiv [Preprint]. 2024 Nov 21:2024.11.20.624542. doi: 10.1101/2024.11.20.624542. bioRxiv. 2024. PMID: 39651281 Free PMC article. Preprint.
Elevation of Granulocyte Colony Stimulating Factor in Human AMD Donor RPE-Choroid.
Mulfaul K, Khan AH, Schwarte SG, Voigt AP, Moore RF, Potempa LA, Wang K, Scheetz TE, Stone EM, Tucker BA, Mullins RF. Mulfaul K, et al. Among authors: stone em. Invest Ophthalmol Vis Sci. 2024 Dec 2;65(14):15. doi: 10.1167/iovs.65.14.15. Invest Ophthalmol Vis Sci. 2024. PMID: 39641748 Free PMC article.
1,610 results