3319-A HOST PRECONDITIONING AND TRANSIENT MITOGEN EXPRESSION VIA mRNA-LNP LEAD TO ROBUST PRIMARY HUMAN HEPATOCYTE ENGRAFTMENT AND TRANSIENT iPSC-DERIVED HEPATOCYTE SURVIVAL IN A MURINE MODEL OF AATD LIVER DISEASE Anna R Smith 1 , Fatima Rizvi 1 , Elissa Everton 1 , Anisah Adeagbo 1 , Hua Liu 1 , Ying Tam 2 , Norbert Pardi 3 , Drew Weissman 3 and Valerie Gouon-Evans 1 , (1)Boston University School of Medicine, (2)Acuitas Therapeutics, (3)University of Pennsylvania Perelman School of Medicine Background: Alpha-1 antitrypsin deficiency (AATD) is a genetic disease caused by a single base pair mutation of the SERPINA1 gene and increases risk of liver and lung disease
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Ginimuge, P.R., & Jyothi, S.D
10.1034/j.1399-3003.2000.016003534.x Eur Respir J