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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Publicado en: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
Formato: Artigo
Idioma:Inglês
Publicado: John Wiley and Sons Inc. 2021
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Acceso en liña: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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