In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions
C. Nicolas, C. Van Lith, K. Allen, R. Hickey, Z. Du, L. Hillin, R. Guthman, W. Cao, A. Bhagwate, D. O'Brien, J.-P. Kocher, R. A. Kaiser, S. Russell, J. Lillegard
This study cures hereditary tyrosinemia type 1 in pigs using in vivo lentiviral gene therapy, preventing liver fibrosis and tumorigenic pathways.
bioRxiv · Jan 2, 2021Cell Biology