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Targeting intracellular degradation pathways for treatment of liver disease caused by α1-antitrypsin deficiency
The classic form of α1-antitrypsin deficiency (ATD) is a well-known genetic cause of severe liver disease in childhood. A point mutation alters the folding of a hepatic secretory glycoprotein such that the protein is prone to misfolding and polymerization. Liver injury, characterized predominantly b...
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| Auteurs principaux: | , |
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| Format: | Artigo |
| Langue: | Inglês |
| Publié: |
2013
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| Sujets: | |
| Accès en ligne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4174576/ https://ncbi.nlm.nih.gov/pubmed/24226634 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/pr.2013.190 |
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