Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1
Author(s) -
Raymond Hickey,
Shennen A. Mao,
Jaime Glorioso,
Faysal Elgilani,
Bruce Amiot,
Harvey Chen,
Piero Rinaldo,
Ronald J. Marler,
Huailei Jiang,
Timothy R. DeGrado,
Lukkana Suksanpaisan,
Michael K. O’Connor,
Brittany L. Freeman,
Samar H. Ibrahim,
Kah Whye Peng,
Cary O. Harding,
ChakSum Ho,
Markus Grompe,
Yasuhiro Ikeda,
Joseph B. Lillegard,
Stephen J. Russell,
Scott L. Nyberg
Publication year - 2016
Publication title -
science translational medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 6.819
H-Index - 216
eISSN - 1946-6242
pISSN - 1946-6234
DOI - 10.1126/scitranslmed.aaf3838
Subject(s) - tyrosinemia , ex vivo , genetic enhancement , liver transplantation , medicine , gene , transplantation , in vivo , liver disease , cancer research , biology , genetics , biochemistry , tyrosine
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