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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7933930/
https://www.ncbi.nlm.nih.gov/pubmed/33512066
http://dx.doi.org/10.15252/emmm.202013167

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7933930/
https://www.ncbi.nlm.nih.gov/pubmed/33512066
http://dx.doi.org/10.15252/emmm.202013167

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