The development of highly potent and selective small molecule correctors of Z α1-antitrypsin misfolding.
Bioorg Med Chem Lett
; 41: 127973, 2021 06 01.
Article
in En
| MEDLINE
| ID: mdl-33753261
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.
Key words
Full text:
1
Database:
MEDLINE
Main subject:
Drug Design
/
Alpha 1-Antitrypsin
/
Protein Folding
Type of study:
Prognostic_studies
Limits:
Humans
Language:
En
Year:
2021
Type:
Article