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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Publicat a:EMBO Mol Med
Autors principals: 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
Idioma:Inglês
Publicat: John Wiley and Sons Inc. 2021
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Articles
Accés en línia: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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