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Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial.
Fischer MD, Michalakis S, Wilhelm B, Zobor D, Muehlfriedel R, Kohl S, Weisschuh N, Ochakovski GA, Klein R, Schoen C, Sothilingam V, Garcia-Garrido M, Kuehlewein L, Kahle N, Werner A, Dauletbekov D, Paquet-Durand F, Tsang S, Martus P, Peters T, Seeliger M, Bartz-Schmidt KU, Ueffing M, Zrenner E, Biel M, Wissinger B. Fischer MD, et al. Among authors: ueffing m. JAMA Ophthalmol. 2020 Jun 1;138(6):643-651. doi: 10.1001/jamaophthalmol.2020.1032. JAMA Ophthalmol. 2020. PMID: 32352493 Free PMC article. Clinical Trial.
Successful subretinal delivery and monitoring of MicroBeads in mice.
Fischer MD, Goldmann T, Wallrapp C, Mühlfriedel R, Beck SC, Stern-Schneider G, Ueffing M, Wolfrum U, Seeliger MW. Fischer MD, et al. Among authors: ueffing m. PLoS One. 2013;8(1):e55173. doi: 10.1371/journal.pone.0055173. Epub 2013 Jan 28. PLoS One. 2013. PMID: 23383096 Free PMC article.
Identification of a common non-apoptotic cell death mechanism in hereditary retinal degeneration.
Arango-Gonzalez B, Trifunović D, Sahaboglu A, Kranz K, Michalakis S, Farinelli P, Koch S, Koch F, Cottet S, Janssen-Bienhold U, Dedek K, Biel M, Zrenner E, Euler T, Ekström P, Ueffing M, Paquet-Durand F. Arango-Gonzalez B, et al. Among authors: ueffing m. PLoS One. 2014 Nov 13;9(11):e112142. doi: 10.1371/journal.pone.0112142. eCollection 2014. PLoS One. 2014. PMID: 25392995 Free PMC article.
Multimodal assessment of choroideremia patients defines pre-treatment characteristics.
Seitz IP, Zhour A, Kohl S, Llavona P, Peter T, Wilhelm B, Zrenner E, Ueffing M, Bartz-Schmidt KU, Fischer MD. Seitz IP, et al. Among authors: ueffing m. Graefes Arch Clin Exp Ophthalmol. 2015 Dec;253(12):2143-50. doi: 10.1007/s00417-015-2976-4. Epub 2015 Mar 7. Graefes Arch Clin Exp Ophthalmol. 2015. PMID: 25744334
HDAC inhibition in the cpfl1 mouse protects degenerating cone photoreceptors in vivo.
Trifunović D, Arango-Gonzalez B, Comitato A, Barth M, Del Amo EM, Kulkarni M, Sahaboglu A, Hauck SM, Urtti A, Arsenijevic Y, Ueffing M, Marigo V, Paquet-Durand F. Trifunović D, et al. Among authors: ueffing m. Hum Mol Genet. 2016 Oct 15;25(20):4462-4472. doi: 10.1093/hmg/ddw275. Hum Mol Genet. 2016. PMID: 28172811 Free article.
AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye.
Reichel FF, Dauletbekov DL, Klein R, Peters T, Ochakovski GA, Seitz IP, Wilhelm B, Ueffing M, Biel M, Wissinger B, Michalakis S, Bartz-Schmidt KU, Fischer MD; RD-CURE Consortium. Reichel FF, et al. Among authors: ueffing m. Mol Ther. 2017 Dec 6;25(12):2648-2660. doi: 10.1016/j.ymthe.2017.08.018. Epub 2017 Aug 31. Mol Ther. 2017. PMID: 28970046 Free PMC article.
Superior Retinal Gene Transfer and Biodistribution Profile of Subretinal Versus Intravitreal Delivery of AAV8 in Nonhuman Primates.
Seitz IP, Michalakis S, Wilhelm B, Reichel FF, Ochakovski GA, Zrenner E, Ueffing M, Biel M, Wissinger B, Bartz-Schmidt KU, Peters T, Fischer MD; RD-CURE Consortium. Seitz IP, et al. Among authors: ueffing m. Invest Ophthalmol Vis Sci. 2017 Nov 1;58(13):5792-5801. doi: 10.1167/iovs.17-22473. Invest Ophthalmol Vis Sci. 2017. PMID: 29117317
330 results