TY  - GEN
AU  - Pastore,Nunzia
AU  - Blomenkamp,Keith
AU  - Annunziata,Fabio
AU  - Piccolo,Pasquale
AU  - Mithbaokar,Pratibha
AU  - Maria Sepe,Rosa
AU  - Vetrini,Francesco
AU  - Palmer,Donna
AU  - Ng,Philip
AU  - Polishchuk,Elena
AU  - Iacobacci,Simona
AU  - Polishchuk,Roman
AU  - Teckman,Jeffrey
AU  - Ballabio,Andrea
AU  - Brunetti-Pierri,Nicola
TI  - Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency
SN  - 1757-4684
PY  - 2013///0722
KW  - Animals
KW  - Apoptosis
KW  - Autophagy
KW  - genetics
KW  - Autophagy-Related Protein 7
KW  - Basic Helix-Loop-Helix Leucine Zipper Transcription Factors
KW  - Disease Models, Animal
KW  - Gene Transfer Techniques
KW  - Genetic Predisposition to Disease
KW  - Genetic Therapy
KW  - methods
KW  - HeLa Cells
KW  - Humans
KW  - Interleukin-6
KW  - metabolism
KW  - Liver
KW  - enzymology
KW  - Liver Cirrhosis
KW  - Lysosomes
KW  - Mice
KW  - Mice, Inbred C57BL
KW  - Mice, Knockout
KW  - Mice, Transgenic
KW  - Microtubule-Associated Proteins
KW  - deficiency
KW  - Mutation, Missense
KW  - NF-kappa B
KW  - Papio
KW  - Phenotype
KW  - Time Factors
KW  - Transfection
KW  - alpha 1-Antitrypsin
KW  - alpha 1-Antitrypsin Deficiency
KW  - complications
N1  - Publication Type: Journal Article; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1002/emmm.201202046
ER  -