Article
Knockdown of Alpha-1 Antitrypsin with antisense oligonucleotide does not exacerbate smoke induced lung injury.
PloS one - 1 Jan 2021
Stearns Kyle, Goldklang Monica, Xiao Rui, Zelonina Tina, Blomenkamp Keith, Teckman Jeffery, D'Armiento Jeanine M
Abstract excerpt
Alpha-1 Antitrypsin (AAT) is a serum protease inhibitor that regulates increased lung protease production induced by cigarette smoking. Mutations in the Serpina1 gene cause AAT to form hepatoxic polymers, which can lead to reduced availability for the protein's primary function and severe liver disease. An AAT antisense oligonucleotide (ASO) was previously identified to be beneficial for the AATD liver disease by...
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