Targeting ABCB4 using mRNA-LNP for the treatment of rare liver diseases
Mohammed Alsuraih, Brianna LaViolette, Guan-Yu Lin, Ramesh C. Kovi, Natalie A. Daurio, Congsheng Cheng, Youngwook Ahn, Zhihua Jiang, Roberto Ortiz, Shangzhong Li, Yuxing Cheng, Ye Wang, Xiaoyu Fan, Jessica Haskins, Xiuhua Sun, , Shian-Huey Chiang
This study demonstrates that systemic delivery of hABCB4 mRNA LNPs ameliorates liver fibrosis and bile phosphatidylcholine levels in a PFIC3 mouse model.
bioRxiv · Apr 11, 2023 · PreprintMolecular Biology