Citations 2
Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1
Mao, Shennen A. , Glorioso, Jaime , Elgilani, Faysal, Amiot, Bruce , Chen, Harvey , Rinaldo, Piero, Marler, Ronald J., Jiang, Huailei , DeGrado, Timothy R. , Suksanpaisan, Lukkana , O’Connor, Michael K. , Freeman, Brittany L., Ibrahim, Samar H. , Peng, Kah Whye, Grompe, Markus , Ikeda, Yasuhiro , Lillegard, Joseph B. , Russell, Stephen J. , Nyberg, Scott L. , Hickey, Raymond D, Marler, Ronald, O'Connor, Michael K, Hickey, Raymond , Harding, Cary O. , Ho, Chak‐Sum , Russell, Stephen J , Ibrahim, Samar H , Mao, Shennen A , Jiang, Huailei
Science Translational Medicine | July 27, 2016 | Volume: 8 | Issue: 349 | Pages: No page available
Transplantation of gene-corrected autologous hepatocytes can cure metabolic disease in a preclinical pig model of hereditary tyrosinemia type 1.
Silent Tyrosinemia Type I Without Elevated Tyrosine or Succinylacetone Associated with Liver Cirrhosis and Hepatocellular Carcinoma
Blackburn, Patrick R. , Nace, Rebecca A., Giama, Nasra H., Kraft, Daniel, Bordner, Andrew J., Graham, Rondell P. , Tortorelli, Silvia , Lindor, Noralane M. , Milliner, Dawn S. , Oglesbee, Devin , Al-Qabandi, Wafa’a, Grompe, Markus , Gavrilov, Dimitar K. , El‐Youssef, Mounif, Clark, Karl J. , Roberts, Lewis R. , Klee, Eric W. , Ekker, Stephen C. , Hickey, Raymond D, Kraft, Daniel L, McCormick, Jennifer B, Radulovic, Maja, El-Youssef, Mounif, Atwal, Paldeep S. , Hickey, Raymond , Chaiteerakij, Roongruedee , McCormick, Jennifer Blair , Radulovic, Maja , Torbenson, Michael , Scott, C. Ronald , Atwal, Paldeep S , Roberts, Lewis R , Ekker, Stephen C , Clark, Karl J , Graham, Rondell P , Oglesbee, Devin
Human Mutation | July 11, 2016 | Volume: 37 | Issue: 10 | Pages: 1097-1105