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Using CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration.
Burnight ER, Gupta M, Wiley LA, Anfinson KR, Tran A, Triboulet R, Hoffmann JM, Klaahsen DL, Andorf JL, Jiao C, Sohn EH, Adur MK, Ross JW, Mullins RF, Daley GQ, Schlaeger TM, Stone EM, Tucker BA. Burnight ER, et al. Mol Ther. 2017 Sep 6;25(9):1999-2013. doi: 10.1016/j.ymthe.2017.05.015. Epub 2017 Jun 12. Mol Ther. 2017. PMID: 28619647 Free PMC article.
CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype.
Burnight ER, Wiley LA, Drack AV, Braun TA, Anfinson KR, Kaalberg EE, Halder JA, Affatigato LM, Mullins RF, Stone EM, Tucker BA. Burnight ER, et al. Gene Ther. 2014 Jul;21(7):662-72. doi: 10.1038/gt.2014.39. Epub 2014 May 8. Gene Ther. 2014. PMID: 24807808 Free PMC article.
Gene therapy using stem cells.
Burnight ER, Wiley LA, Mullins RF, Stone EM, Tucker BA. Burnight ER, et al. Cold Spring Harb Perspect Med. 2014 Nov 13;5(4):a017434. doi: 10.1101/cshperspect.a017434. Cold Spring Harb Perspect Med. 2014. PMID: 25395375 Free PMC article. Review.
Concise Review: Patient-Specific Stem Cells to Interrogate Inherited Eye Disease.
Giacalone JC, Wiley LA, Burnight ER, Songstad AE, Mullins RF, Stone EM, Tucker BA. Giacalone JC, et al. Among authors: burnight er. Stem Cells Transl Med. 2016 Feb;5(2):132-40. doi: 10.5966/sctm.2015-0206. Epub 2015 Dec 18. Stem Cells Transl Med. 2016. PMID: 26683869 Free PMC article. Review.
Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3-Associated Retinal Degeneration.
Wiley LA, Burnight ER, Drack AV, Banach BB, Ochoa D, Cranston CM, Madumba RA, East JS, Mullins RF, Stone EM, Tucker BA. Wiley LA, et al. Among authors: burnight er. Hum Gene Ther. 2016 Oct;27(10):835-846. doi: 10.1089/hum.2016.049. Epub 2016 Jul 11. Hum Gene Ther. 2016. PMID: 27400765 Free PMC article.
Assessment of Adeno-Associated Virus Serotype Tropism in Human Retinal Explants.
Wiley LA, Burnight ER, Kaalberg EE, Jiao C, Riker MJ, Halder JA, Luse MA, Han IC, Russell SR, Sohn EH, Stone EM, Tucker BA, Mullins RF. Wiley LA, et al. Among authors: burnight er. Hum Gene Ther. 2018 Apr;29(4):424-436. doi: 10.1089/hum.2017.179. Epub 2018 Feb 23. Hum Gene Ther. 2018. PMID: 29160116 Free PMC article.
31 results