Alpha1-Antitrypsin Deficiency and Autophagy
SJ Marciniak, DA Lomas - New England Journal of Medicine, 2010 - Mass Medical Soc
New England Journal of Medicine, 2010•Mass Medical Soc
The Z deficiency allele (PI* Z) of SERPINA1, which encodes the serine protease inhibitor (PI)
alpha1-antitrypsin, results in the formation of intrahepatic inclusions that cause cirrhosis. The
only curative therapy for patients with severe liver disease is transplantation. Hidvegi and
colleagues recently described a new approach to clear the pathologic inclusions in cell and
animal models of liver disease consequent to the Z mutation. The authors used the
antiepileptic drug carbamazepine to stimulate “self-eating,” or autophagy. 1 The questions …
alpha1-antitrypsin, results in the formation of intrahepatic inclusions that cause cirrhosis. The
only curative therapy for patients with severe liver disease is transplantation. Hidvegi and
colleagues recently described a new approach to clear the pathologic inclusions in cell and
animal models of liver disease consequent to the Z mutation. The authors used the
antiepileptic drug carbamazepine to stimulate “self-eating,” or autophagy. 1 The questions …
The Z deficiency allele (PI*Z) of SERPINA1, which encodes the serine protease inhibitor (PI) alpha1-antitrypsin, results in the formation of intrahepatic inclusions that cause cirrhosis. The only curative therapy for patients with severe liver disease is transplantation. Hidvegi and colleagues recently described a new approach to clear the pathologic inclusions in cell and animal models of liver disease consequent to the Z mutation. The authors used the antiepileptic drug carbamazepine to stimulate “self-eating,” or autophagy.1 The questions now are whether this approach will be useful for treating the liver disease associated with alpha1-antitrypsin . . .
The New England Journal Of Medicine