Article

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

2021-09-17

Abstract excerpt

Alpha-1 antitrypsin deficiency is a life-threatening condition caused by inheritance of the SERPINA1 gene ‘Z’ variant. This single base pair mutation leads to protein misfolding, ER entrapment and gain of toxic function. Despite the significant unmet medical need presented by this disorder, there remain no approved medicines and the only curative option is liver transplantation. We hypothesized that an unbiased s...

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Literature Corpus work: 3a73a3bc-943f-5b99-b02d-dfa4699e731c
DOI: 10.1101/2021.09.17.460732

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