Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients

Turner, A.M.; Stolk, J.; Bals, R.; Lickliter, J.D.; Hamilton, J.; Christianson, D.R.; ... ; Teckman, J.H. (2018)

Article / Letter to editor

• siRNA
• Phase 1
• PiZZ
• RNAi
• Therapeutic

• All authors: Turner, A.M.; Stolk, J.; Bals, R.; Lickliter, J.D.; Hamilton, J.; Christianson, D.R.; Given, B.D.; Burdon, J.G.; Loomba, R.; Stoller, J.K.; Teckman, J.H.
• Date: 2018-08-31
• Journal: Journal of Hepatology
• Volume: 69
• Issue: 2
• Pages: 378 - 384
• DOI: doi:10.1016/j.jhep.2018.03.012