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Impaired ABCA1/ABCG1-mediated lipid efflux in the mouse retinal pigment epithelium (RPE) leads to retinal degeneration.
Storti F, Klee K, Todorova V, Steiner R, Othman A, van der Velde-Visser S, Samardzija M, Meneau I, Barben M, Karademir D, Pauzuolyte V, Boye SL, Blaser F, Ullmer C, Dunaief JL, Hornemann T, Rohrer L, den Hollander A, von Eckardstein A, Fingerle J, Maugeais C, Grimm C. Storti F, et al. Among authors: boye sl. Elife. 2019 Mar 13;8:e45100. doi: 10.7554/eLife.45100. Elife. 2019. PMID: 30864945 Free PMC article.
Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.
Beltran WA, Cideciyan AV, Iwabe S, Swider M, Kosyk MS, McDaid K, Martynyuk I, Ying GS, Shaffer J, Deng WT, Boye SL, Lewin AS, Hauswirth WW, Jacobson SG, Aguirre GD. Beltran WA, et al. Among authors: boye sl. Proc Natl Acad Sci U S A. 2015 Oct 27;112(43):E5844-53. doi: 10.1073/pnas.1509914112. Epub 2015 Oct 12. Proc Natl Acad Sci U S A. 2015. PMID: 26460017 Free PMC article.
AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy.
Dinculescu A, Stupay RM, Deng WT, Dyka FM, Min SH, Boye SL, Chiodo VA, Abrahan CE, Zhu P, Li Q, Strettoi E, Novelli E, Nagel-Wolfrum K, Wolfrum U, Smith WC, Hauswirth WW. Dinculescu A, et al. Among authors: boye sl. PLoS One. 2016 Feb 16;11(2):e0148874. doi: 10.1371/journal.pone.0148874. eCollection 2016. PLoS One. 2016. PMID: 26881841 Free PMC article.
123 results