Back to Authors

Marler, Ronald

Mayo Clinic

Department of Comparative Medicine, Mayo Clinic, Scottsdale, AZ 85259, USA.

Citations 1

Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1

Science Translational Medicine | July 27, 2016 | Volume: 8 | Issue: 349 | Pages: No page available

10.1126/scitranslmed.aaf3838 | PMID 27464750

Transplantation of gene-corrected autologous hepatocytes can cure metabolic disease in a preclinical pig model of hereditary tyrosinemia type 1.