Article

Development of a small molecule that corrects misfolding and increases secretion of Z α <sub>1</sub> -antitrypsin

2020-07-28

Abstract excerpt

Severe α 1 -antitrypsin deficiency results from the Z allele (Glu342Lys) that causes the accumulation of homopolymers of mutant α 1 -antitrypsin within the endoplasmic reticulum of hepatocytes in association with liver disease. We have used a DNA-encoded chemical library to undertake a high throughput screen to identify small molecules that bind to, and stabilise Z α 1 -antitrypsin. The lead compound blocks Z α...

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Literature Corpus work: 828fdb4e-2c9d-5372-80e0-64c1f8d8ae28
DOI: 10.1101/2020.07.26.217661

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