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Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR.
Cehajic-Kapetanovic J, Xue K, Martinez-Fernandez de la Camara C, Nanda A, Davies A, Wood LJ, Salvetti AP, Fischer MD, Aylward JW, Barnard AR, Jolly JK, Luo E, Lujan BJ, Ong T, Girach A, Black GCM, Gregori NZ, Davis JL, Rosa PR, Lotery AJ, Lam BL, Stanga PE, MacLaren RE. Cehajic-Kapetanovic J, et al. Among authors: maclaren re. Nat Med. 2020 Mar;26(3):354-359. doi: 10.1038/s41591-020-0763-1. Epub 2020 Feb 24. Nat Med. 2020. PMID: 32094925 Free PMC article. Clinical Trial.
Effective gene therapy with nonintegrating lentiviral vectors.
Yáñez-Muñoz RJ, Balaggan KS, MacNeil A, Howe SJ, Schmidt M, Smith AJ, Buch P, MacLaren RE, Anderson PN, Barker SE, Duran Y, Bartholomae C, von Kalle C, Heckenlively JR, Kinnon C, Ali RR, Thrasher AJ. Yáñez-Muñoz RJ, et al. Among authors: maclaren re. Nat Med. 2006 Mar;12(3):348-53. doi: 10.1038/nm1365. Epub 2006 Feb 19. Nat Med. 2006. PMID: 16491086
Characterization of a dominant cone degeneration in a green fluorescent protein-reporter mouse with disruption of Loci associated with human dominant retinal dystrophy.
Lipinski DM, Yusuf M, Barnard AR, Damant C, Charbel Issa P, Singh MS, Lee E, Davies WL, Volpi EV, MacLaren RE. Lipinski DM, et al. Among authors: maclaren re. Invest Ophthalmol Vis Sci. 2011 Aug 22;52(9):6617-23. doi: 10.1167/iovs.11-7932. Invest Ophthalmol Vis Sci. 2011. PMID: 21705682 Free PMC article.
Clinical applications of retinal gene therapy.
Lipinski DM, Thake M, MacLaren RE. Lipinski DM, et al. Among authors: maclaren re. Prog Retin Eye Res. 2013 Jan;32:22-47. doi: 10.1016/j.preteyeres.2012.09.001. Epub 2012 Sep 17. Prog Retin Eye Res. 2013. PMID: 22995954 Review.
Gene therapy for retinal disease.
McClements ME, MacLaren RE. McClements ME, et al. Among authors: maclaren re. Transl Res. 2013 Apr;161(4):241-54. doi: 10.1016/j.trsl.2012.12.007. Epub 2013 Jan 8. Transl Res. 2013. PMID: 23305707 Free PMC article. Review.
357 results