Development of a small molecule that corrects misfolding and increases secretion of Z α1‐antitrypsin

Development of a small molecule that corrects misfolding and increases secretion of Z α1‐antitrypsin

Abstract Severe α1‐antitrypsin deficiency results from the Z allele (Glu342Lys) that causes the accumulation of homopolymers of mutant α1‐antitrypsin within the endoplasmic reticulum of hepatocytes in association with liver disease. We have used a DNA‐encoded chemical library to undertake a high‐thr...

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Bibliographic Details
Main Authors: David A Lomas, James A Irving, Christopher Arico‐Muendel, Svetlana Belyanskaya, Andrew Brewster, Murray Brown, Chun‐wa Chung, Hitesh Dave, Alexis Denis, Nerina Dodic, Anthony Dossang, Peter Eddershaw, Diana Klimaszewska, Imran Haq, Duncan S Holmes, Jonathan P Hutchinson, Alistair M Jagger, Toral Jakhria, Emilie Jigorel, John Liddle, Ken Lind, Stefan J Marciniak, Jeff Messer, Margaret Neu, Allison Olszewski, Adriana Ordonez, Riccardo Ronzoni, James Rowedder, Martin Rüdiger, Steve Skinner, Kathrine J Smith, Rebecca Terry, Lionel Trottet, Iain Uings, Steve Wilson, Zhengrong Zhu, Andrew C Pearce
Format: Article
Language:English
Published: Wiley 2021-03-01
Series:EMBO Molecular Medicine
Subjects:
emphysema
liver disease
protein misfolding
small molecule corrector
α1‐antitrypsin deficiency
Online Access:https://doi.org/10.15252/emmm.202013167

Internet

https://doi.org/10.15252/emmm.202013167

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