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Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model.
Hsu Y, Bhattarai S, Thompson JM, Mahoney A, Thomas J, Mayer SK, Datta P, Garrison J, Searby CC, Vandenberghe LH, Seo S, Sheffield VC, Drack AV. Hsu Y, et al. Among authors: vandenberghe lh. Mol Ther Nucleic Acids. 2022 Dec 12;31:164-181. doi: 10.1016/j.omtn.2022.12.007. eCollection 2023 Mar 14. Mol Ther Nucleic Acids. 2022. PMID: 36700052 Free PMC article.
Breaking and Sealing Barriers in Retinal Gene Therapy.
Miller JW, Vandenberghe LH. Miller JW, et al. Among authors: vandenberghe lh. Mol Ther. 2018 Sep 5;26(9):2081-2082. doi: 10.1016/j.ymthe.2018.08.003. Epub 2018 Aug 11. Mol Ther. 2018. PMID: 30107998 Free PMC article. No abstract available.
Early disruption of photoreceptor cell architecture and loss of vision in a humanized pig model of usher syndromes.
Grotz S, Schäfer J, Wunderlich KA, Ellederova Z, Auch H, Bähr A, Runa-Vochozkova P, Fadl J, Arnold V, Ardan T, Veith M, Santamaria G, Dhom G, Hitzl W, Kessler B, Eckardt C, Klein J, Brymova A, Linnert J, Kurome M, Zakharchenko V, Fischer A, Blutke A, Döring A, Suchankova S, Popelar J, Rodríguez-Bocanegra E, Dlugaiczyk J, Straka H, May-Simera H, Wang W, Laugwitz KL, Vandenberghe LH, Wolf E, Nagel-Wolfrum K, Peters T, Motlik J, Fischer MD, Wolfrum U, Klymiuk N. Grotz S, et al. Among authors: vandenberghe lh. EMBO Mol Med. 2022 Apr 7;14(4):e14817. doi: 10.15252/emmm.202114817. Epub 2022 Mar 7. EMBO Mol Med. 2022. PMID: 35254721 Free PMC article.
Promising and delivering gene therapies for vision loss.
Carvalho LS, Vandenberghe LH. Carvalho LS, et al. Among authors: vandenberghe lh. Vision Res. 2015 Jun;111(Pt B):124-33. doi: 10.1016/j.visres.2014.07.013. Epub 2014 Aug 2. Vision Res. 2015. PMID: 25094052 Free PMC article. Review.
What Is Next for Retinal Gene Therapy?
Vandenberghe LH. Vandenberghe LH. Cold Spring Harb Perspect Med. 2015 Apr 15;5(10):a017442. doi: 10.1101/cshperspect.a017442. Cold Spring Harb Perspect Med. 2015. PMID: 25877395 Free PMC article. Review.
109 results