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MODIFIED MRNA FOR THE TREATMENT OF PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS DISORDERS
MODIFIED MRNA FOR THE TREATMENT OF PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS DISORDERS
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机译:修饰mRNA用于治疗进步性家族性肝内胆囊疾病
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摘要
The present disclosure provides compositions of nucleic acids relating to biliary epithelial transporters. For example, the present disclosure relates to nucleic acids capable of regulating the biliary secretion of phospholipids, including phosphatidylcholine, e.g., those encoded by ATP binding cassette subfamily B member 4 (ABCB4) or a biologically active fragment thereof, in a target cell. In a preferred embodiment, the present disclosure provides compositions comprising modified mRNA encoding ABCB4 formulated in a lipid nanoparticle (LNP) carrier and derivative constructs, which are useful for treating or preventing progressive familial intrahepatic cholestasis type 3 (PFIC3).
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