The role of autophagy in alpha-1-antitrypsin deficiency
T Hidvegi, A Mukherjee, M Ewing, C Kemp… - Methods in …, 2011 - Elsevier
In the classical form of alpha-1-antitrypsin (AT) deficiency, a mutant protein accumulates in
the endoplasmic reticulum of liver cells, causing hepatic fibrosis and hepatocellular
carcinoma by a gain-of-toxic function mechanism. Autophagy is specifically activated by the
accumulation of mutant AT, and the autophagy plays a key role in intracellular degradation
of this mutant protein. Our recent study indicates that an autophagy enhancer drug can
decrease the hepatic load of mutant AT and reduce hepatic fibrosis in a mouse model of AT …
the endoplasmic reticulum of liver cells, causing hepatic fibrosis and hepatocellular
carcinoma by a gain-of-toxic function mechanism. Autophagy is specifically activated by the
accumulation of mutant AT, and the autophagy plays a key role in intracellular degradation
of this mutant protein. Our recent study indicates that an autophagy enhancer drug can
decrease the hepatic load of mutant AT and reduce hepatic fibrosis in a mouse model of AT …