Development of a small molecule that corrects misfolding and increases secretion of Z α(1)‐antitrypsin

Severe α(1)‐antitrypsin deficiency results from the Z allele (Glu342Lys) that causes the accumulation of homopolymers of mutant α(1)‐antitrypsin within the endoplasmic reticulum of hepatocytes in association with liver disease. We have used a DNA‐encoded chemical library to undertake a high‐throughp...

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Published in:EMBO Mol Med
Main Authors: Lomas, David A, Irving, James A, Arico‐Muendel, Christopher, Belyanskaya, Svetlana, Brewster, Andrew, Brown, Murray, Chung, Chun‐wa, Dave, Hitesh, Denis, Alexis, Dodic, Nerina, Dossang, Anthony, Eddershaw, Peter, Klimaszewska, Diana, Haq, Imran, Holmes, Duncan S, Hutchinson, Jonathan P, Jagger, Alistair M, Jakhria, Toral, Jigorel, Emilie, Liddle, John, Lind, Ken, Marciniak, Stefan J, Messer, Jeff, Neu, Margaret, Olszewski, Allison, Ordonez, Adriana, Ronzoni, Riccardo, Rowedder, James, Rüdiger, Martin, Skinner, Steve, Smith, Kathrine J, Terry, Rebecca, Trottet, Lionel, Uings, Iain, Wilson, Steve, Zhu, Zhengrong, Pearce, Andrew C
Format: Artigo
Language:Inglês
Published: John Wiley and Sons Inc. 2021
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC7933930/
https://ncbi.nlm.nih.gov/pubmed/33512066
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.202013167
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