Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency.

Pastore, Nunzia

Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency. [electronic resource] - EMBO molecular medicine Mar 2013 - 397-412 p. digital

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

1757-4684

10.1002/emmm.201202046 doi


Animals
Apoptosis
Autophagy--genetics
Autophagy-Related Protein 7
Basic Helix-Loop-Helix Leucine Zipper Transcription Factors--genetics
Disease Models, Animal
Gene Transfer Techniques
Genetic Predisposition to Disease
Genetic Therapy--methods
HeLa Cells
Humans
Interleukin-6--metabolism
Liver--enzymology
Liver Cirrhosis--genetics
Lysosomes--metabolism
Mice
Mice, Inbred C57BL
Mice, Knockout
Mice, Transgenic
Microtubule-Associated Proteins--deficiency
Mutation, Missense
NF-kappa B--metabolism
Papio
Phenotype
Time Factors
Transfection
alpha 1-Antitrypsin--genetics
alpha 1-Antitrypsin Deficiency--complications