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...

ver descrição completa

Na minha lista:
Detalhes bibliográficos
Publicado no: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.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2020
Assuntos:
Research Article
Acesso em linha: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
Tags: Adicionar Tag
Sem tags, seja o primeiro a adicionar uma tag!

Registos relacionados