Reduction of Z alpha-1 antitrypsin polymers in human iPSC-hepatocytes and mice by LRRK2 inhibitors.
Deniz KentSoon Seng NgAdam M SyandaPayam KhoshkenarRiccardo RonzoniChao Zheng LiMarina ZiegerCindy GreerStephanie HatchJoe SegalSamuel J I BlackfordYu Ri ImVivek ChowdaryTaylor IsmailiDavide DanoviPatrick A LewisJames A IrvingSunil SahdeoDavid A LomasDaniel EbnerChristian MuellerSheikh Tamir RashidPublished in: Hepatology (Baltimore, Md.) (2024)
Our findings support the use of CZC-25146 and leucine-rich repeat kinase-2 inhibitors in hepatic proteinopathy research and their further investigation as novel therapeutic candidates for A1ATD.