Article

Small molecule screen employing patient-derived iPSC hepatocytes identifies LRRK2 as a novel therapeutic target for Alpha1 Antitrypsin Deficiency

2022-02-02

Abstract excerpt

<title>Abstract</title> Alpha-1 antitrypsin deficiency (A1ATD) is a life-threatening condition caused by inheritance of the SERPINA1-Z genetic variant (PiZ) leading to protein misfolding and liver toxicity. There remain no approved medicines for this disease. Here we report the results of a small molecule screen performed in patient-derived iPSC hepatocytes that identified Leucine-rich repeat kinase-2 (LRRK2),...

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Literature Corpus work: 7568823d-7ce2-5681-8eaf-2523719d1c04
DOI: 10.21203/rs.3.rs-1283764/v1

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