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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: surace em. 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: surace em. Mol Ther. 2004 Feb;9(2):182-8. doi: 10.1016/j.ymthe.2003.11.013. Mol Ther. 2004. PMID: 14759802 Free article.
Gene therapy for Leber congenital amaurosis.
Dejneka NS, Surace EM, Bennett J. Dejneka NS, et al. Among authors: surace em. Adv Exp Med Biol. 2003;533:415-22. doi: 10.1007/978-1-4615-0067-4_53. Adv Exp Med Biol. 2003. PMID: 15180293 Review. No abstract available.
63 results