Antisense oligonucleotide treatment ameliorates alpha-1 antitrypsin–related liver disease in mice

Alpha-1 antitrypsin deficiency (AATD) is a rare genetic disease that results from mutations in the alpha-1 antitrypsin (AAT) gene. The mutant AAT protein aggregates and accumulates in the liver leading to AATD liver disease, which is only treatable by liver transplant. The PiZ transgenic mouse strai...

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Main Authors: Guo, Shuling, Booten, Sheri L., Aghajan, Mariam, Hung, Gene, Zhao, Chenguang, Blomenkamp, Keith, Gattis, Danielle, Watt, Andrew, Freier, Susan M., Teckman, Jeffery H., McCaleb, Michael L., Monia, Brett P.
Formato: Artigo
Idioma:Inglês
Publicado: American Society for Clinical Investigation 2013
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Research Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3871221/
https://ncbi.nlm.nih.gov/pubmed/24355919
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI67968
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