1.
Bioorg Med Chem Lett
; 41: 127973, 2021 06 01.
Artigo
em Inglês
| MEDLINE
| ID: mdl-33753261
RESUMO
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.