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Page 1
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: lyubarsky a. 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.
In utero gene therapy rescues vision in a murine model of congenital blindness.
Dejneka NS, Surace EM, Aleman TS, Cideciyan AV, Lyubarsky A, Savchenko A, Redmond TM, Tang W, Wei Z, Rex TS, Glover E, Maguire AM, Pugh EN Jr, Jacobson SG, Bennett J. Dejneka NS, et al. Among authors: lyubarsky a. Mol Ther. 2004 Feb;9(2):182-8. doi: 10.1016/j.ymthe.2003.11.013. Mol Ther. 2004. PMID: 14759802 Free article.
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: lyubarsky a. 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.
Delivery of adeno-associated virus vectors to the fetal retina: impact of viral capsid proteins on retinal neuronal progenitor transduction.
Surace EM, Auricchio A, Reich SJ, Rex T, Glover E, Pineles S, Tang W, O'Connor E, Lyubarsky A, Savchenko A, Pugh EN Jr, Maguire AM, Wilson JM, Bennett J. Surace EM, et al. Among authors: lyubarsky a. J Virol. 2003 Jul;77(14):7957-63. doi: 10.1128/jvi.77.14.7957-7963.2003. J Virol. 2003. PMID: 12829835 Free PMC article.
Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness.
Song JY, Aravand P, Nikonov S, Leo L, Lyubarsky A, Bennicelli JL, Pan J, Wei Z, Shpylchak I, Herrera P, Bennett DJ, Commins N, Maguire AM, Pham J, den Hollander AI, Cremers FPM, Koenekoop RK, Roepman R, Nishina P, Zhou S, Pan W, Ying GS, Aleman TS, de Melo J, McNamara I, Sun J, Mills J, Bennett J. Song JY, et al. Among authors: lyubarsky a. Mol Ther. 2018 Jun 6;26(6):1581-1593. doi: 10.1016/j.ymthe.2018.03.015. Epub 2018 Mar 21. Mol Ther. 2018. PMID: 29673930 Free PMC article.
RP1 is required for the correct stacking of outer segment discs.
Liu Q, Lyubarsky A, Skalet JH, Pugh EN Jr, Pierce EA. Liu Q, et al. Among authors: lyubarsky a. Invest Ophthalmol Vis Sci. 2003 Oct;44(10):4171-83. doi: 10.1167/iovs.03-0410. Invest Ophthalmol Vis Sci. 2003. PMID: 14507858 Free PMC article.
43 results