Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1.
Hickey RD, Mao SA, Glorioso J, Elgilani F, Amiot B, Chen H, Rinaldo P, Marler R, Jiang H, DeGrado TR, Suksanpaisan L, O'Connor MK, Freeman BL, Ibrahim SH, Peng KW, Harding CO, Ho CS, Grompe M, Ikeda Y, Lillegard JB, Russell SJ, Nyberg SL.
Hickey RD, et al. Among authors: mao sa.
Sci Transl Med. 2016 Jul 27;8(349):349ra99. doi: 10.1126/scitranslmed.aaf3838.
Sci Transl Med. 2016.
PMID: 27464750
Free PMC article.