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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.
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.