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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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Detalhes bibliográficos
Main Authors: Wang, Yan, Perlmutter, David H.
Formato: Artigo
Idioma:Inglês
Publicado em: 2013
Assuntos:
Acesso em linha: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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