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. Among authors: tucker ba.
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.