Liddle, J;
Pearce, AC;
Arico-Muendel, C;
Belyanskaya, S;
Brewster, A;
Brown, M;
Chung, C-W;
... Lomas, DA; + view all
(2021)
The development of highly potent and selective small molecule correctors of Z α1-antitrypsin misfolding.
Bioorganic & Medicinal Chemistry Letters
, 41
, Article 127973. 10.1016/j.bmcl.2021.127973.
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Abstract
α1-antitrypsin deficiency is characterised by the misfolding and intracellular polymerisation of mutant α1-antitrypsin protein within the endoplasmic reticulum (ER) of hepatocytes. Small molecules that bind and stabilise Z α1-antitrypsin were identified via a DNA-encoded library screen. A subsequent structure based optimisation led to a series of highly potent, selective and cellular active α1-antitrypsin correctors.
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