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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...
Tallennettuna:
| Päätekijät: | , , , , , , , , , , , |
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| Aineistotyyppi: | Artigo |
| Kieli: | Inglês |
| Julkaistu: |
American Society for Clinical Investigation
2013
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| Aiheet: | |
| Linkit: | 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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