Development of an RNAi therapeutic for alpha-1-antitrypsin liver disease

The autosomal codominant genetic disorder alpha-1 antitrypsin (AAT) deficiency (AATD) causes pulmonary and liver disease. Individuals homozygous for the mutant Z allele accumulate polymers of Z-AAT protein in hepatocytes, where AAT is primarily produced. This accumulation causes endoplasmic reticulu...

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Udgivet i:JCI Insight
Main Authors: Wooddell, Christine I., Blomenkamp, Keith, Peterson, Ryan M., Subbotin, Vladimir M., Schwabe, Christian, Hamilton, James, Chu, Qili, Christianson, Dawn R., Hegge, Julia O., Kolbe, John, Hamilton, Holly L., Branca-Afrazi, Maria F., Given, Bruce D., Lewis, David L., Gane, Edward, Kanner, Steven B., Teckman, Jeffrey H.
Format: Artigo
Sprog:Inglês
Udgivet: American Society for Clinical Investigation 2020
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7406265/
https://ncbi.nlm.nih.gov/pubmed/32379724
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.135348
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