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AAV2 gene therapy readministration in three adults with congenital blindness.
Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, McCague S, Pierce EA, Chen Y, Bennicelli JL, Zhu X, Ying GS, Sun J, Wright JF, Auricchio A, Simonelli F, Shindler KS, Mingozzi F, High KA, Maguire AM. Bennett J, et al. Among authors: chung dc. Sci Transl Med. 2012 Feb 8;4(120):120ra15. doi: 10.1126/scitranslmed.3002865. Sci Transl Med. 2012. PMID: 22323828 Free PMC article. Clinical Trial.
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.
Bennicelli J, Wright JF, Komaromy A, Jacobs JB, Hauck B, Zelenaia O, Mingozzi F, Hui D, Chung D, Rex TS, Wei Z, Qu G, Zhou S, Zeiss C, Arruda VR, Acland GM, Dell'Osso LF, High KA, Maguire AM, Bennett J. Bennicelli J, et al. Mol Ther. 2008 Mar;16(3):458-65. doi: 10.1038/sj.mt.6300389. Epub 2008 Jan 22. Mol Ther. 2008. PMID: 18209734 Free PMC article.
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.
Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J, Elci OU, Chung DC, Sun J, Wright JF, Cross DR, Aravand P, Cyckowski LL, Bennicelli JL, Mingozzi F, Auricchio A, Pierce EA, Ruggiero J, Leroy BP, Simonelli F, High KA, Maguire AM. Bennett J, et al. Among authors: chung dc. Lancet. 2016 Aug 13;388(10045):661-72. doi: 10.1016/S0140-6736(16)30371-3. Epub 2016 Jun 30. Lancet. 2016. PMID: 27375040 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: chung dc. 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.
The human visual cortex responds to gene therapy-mediated recovery of retinal function.
Ashtari M, Cyckowski LL, Monroe JF, Marshall KA, Chung DC, Auricchio A, Simonelli F, Leroy BP, Maguire AM, Shindler KS, Bennett J. Ashtari M, et al. Among authors: chung dc. J Clin Invest. 2011 Jun;121(6):2160-8. doi: 10.1172/JCI57377. Epub 2011 May 23. J Clin Invest. 2011. PMID: 21606598 Free PMC article. Clinical Trial.
Recent advances in ocular gene therapy.
Chung DC, Lee V, Maguire AM. Chung DC, et al. Curr Opin Ophthalmol. 2009 Sep;20(5):377-81. doi: 10.1097/ICU.0b013e32832f802a. Curr Opin Ophthalmol. 2009. PMID: 19667988 Review.
Gene delivery to the retina: from mouse to man.
Bennett J, Chung DC, Maguire A. Bennett J, et al. Among authors: chung dc. Methods Enzymol. 2012;507:255-74. doi: 10.1016/B978-0-12-386509-0.00013-2. Methods Enzymol. 2012. PMID: 22365778 Free PMC article.
AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models.
Vasireddy V, Mills JA, Gaddameedi R, Basner-Tschakarjan E, Kohnke M, Black AD, Alexandrov K, Zhou S, Maguire AM, Chung DC, Mac H, Sullivan L, Gadue P, Bennicelli JL, French DL, Bennett J. Vasireddy V, et al. Among authors: chung dc. PLoS One. 2013 May 7;8(5):e61396. doi: 10.1371/journal.pone.0061396. Print 2013. PLoS One. 2013. PMID: 23667438 Free PMC article.
281 results