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The development of highly potent and selective small molecule correctors of Z α1-antitrypsin misfolding

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 10.1016/j.bmcl.2021.127973. (In press).

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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.

Type: Article
Title: The development of highly potent and selective small molecule correctors of Z α1-antitrypsin misfolding
Location: England
DOI: 10.1016/j.bmcl.2021.127973
Publisher version: https://doi.org/10.1016/j.bmcl.2021.127973
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: 2-oxindole, Misfolding, α1-antitrypsin
UCL classification: UCL
UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Respiratory Medicine
UCL > Provost and Vice Provost Offices > VP: Health
URI: https://discovery.ucl.ac.uk/id/eprint/10125364
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