4.7 Article

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

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Review Cell Biology

Guidelines for the use and interpretation of assays for monitoring autophagy

Daniel J. Klionsky et al.

AUTOPHAGY (2012)

Article Biochemistry & Molecular Biology

A lysosome-to-nucleus signalling mechanism senses and regulates the lysosome via mTOR and TFEB

Carmine Settembre et al.

EMBO JOURNAL (2012)

Article Gastroenterology & Hepatology

Preventing and reversing the cellular consequences of Z alpha-1 antitrypsin accumulation by targeting s4A

Sam Alam et al.

JOURNAL OF HEPATOLOGY (2012)

Article Cell Biology

Transcriptional Activation of Lysosomal Exocytosis Promotes Cellular Clearance

Diego L. Medina et al.

DEVELOPMENTAL CELL (2011)

Article Biochemistry & Molecular Biology

DJ-1 acts in parallel to the PINK1/parkin pathway to control mitochondrial function and autophagy

Kelly Jean Thomas et al.

HUMAN MOLECULAR GENETICS (2011)

Review Biochemistry & Molecular Biology

Helper-dependent adenoviral vectors for liver-directed gene therapy

Nicola Brunetti-Pierri et al.

HUMAN MOLECULAR GENETICS (2011)

Article Biochemistry & Molecular Biology

Parkin mediates beclin-dependent autophagic clearance of defective mitochondria and ubiquitinated Aβ in AD models

Preeti J. Khandelwal et al.

HUMAN MOLECULAR GENETICS (2011)

Article Medicine, General & Internal

Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B

Amit C. Nathwani et al.

NEW ENGLAND JOURNAL OF MEDICINE (2011)

Article Multidisciplinary Sciences

TFEB Links Autophagy to Lysosomal Biogenesis

Carmine Settembre et al.

SCIENCE (2011)

Article Medicine, Research & Experimental

Rapamycin reduces intrahepatic alpha-1-antitrypsin mutant Z protein polymers and liver injury in a mouse model

Shalesh Kaushal et al.

EXPERIMENTAL BIOLOGY AND MEDICINE (2010)

Article Virology

Chromosomal Integration of Adenoviral Vector DNA In Vivo

Sam Laurel Stephen et al.

JOURNAL OF VIROLOGY (2010)

Review Genetics & Heredity

Progress Towards Liver and Lung-Directed Gene Therapy with Helper-Dependent Adenoviral Vectors

Nicola Brunetti-Pierri et al.

CURRENT GENE THERAPY (2009)

Review Cell Biology

Lysosome biogenesis and lysosomal membrane proteins: trafficking meets function

Paul Saftig et al.

NATURE REVIEWS MOLECULAR CELL BIOLOGY (2009)

Article Multidisciplinary Sciences

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Mark L. Brantly et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)

Article Medicine, Research & Experimental

In vivo post-transcriptional gene silencing of α-1 antitrypsin by adeno-associated virus vectors expressing siRNA

Pedro E. Cruz et al.

LABORATORY INVESTIGATION (2007)

Article Biochemistry & Molecular Biology

p62/SQSTM1 binds directly to Atg8/LC3 to facilitate degradation of ubiquitinated protein aggregates by autophagy

Serhiy Pankiv et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2007)

Article Biotechnology & Applied Microbiology

Pseudo-hydrodynamic delivery of helper-dependent adenoviral vectors into non-human primates for liver-directed gene therapy

Nicola Brunetti-Pierri et al.

MOLECULAR THERAPY (2007)

Article Gastroenterology & Hepatology

High prevalence of α-1-antitrypsin heterozygosity in children with chronic liver disease

Kathleen M. Campbell et al.

JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION (2007)

Article Biochemistry & Molecular Biology

Intracellular inclusions containing mutant α1-antitrypsin Z are propagated in the absence of autophagic activity

T Kamimoto et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2006)

Article Biotechnology & Applied Microbiology

Sustained phenotypic correction of canine hemophilia B after systemic administration of helper-dependent adenoviral vector

N Brunetti-Pierri et al.

HUMAN GENE THERAPY (2005)

Article Cell Biology

Impairment of starvation-induced and constitutive autophagy in Atg7-deficient mice

M Komatsu et al.

JOURNAL OF CELL BIOLOGY (2005)

Article Multidisciplinary Sciences

Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector

G Toietta et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)

Article Gastroenterology & Hepatology

Gene therapy for human α1-antitrypsin deficiency in an animal model using SV40-derived vectors

YY Duan et al.

GASTROENTEROLOGY (2004)

Article Gastroenterology & Hepatology

Mitochondrial autophagy and injury in the liver in α1-antitrypsin deficiency

JH Teckman et al.

AMERICAN JOURNAL OF PHYSIOLOGY-GASTROINTESTINAL AND LIVER PHYSIOLOGY (2004)

Article Biotechnology & Applied Microbiology

Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates

N Brunetti-Pierri et al.

HUMAN GENE THERAPY (2004)

Article Biotechnology & Applied Microbiology

Improved system for helper-dependent adenoviral vector production

D Palmer et al.

MOLECULAR THERAPY (2003)

Article Gastroenterology & Hepatology

Fasting in α1-antitrypsin deficient liver:: consultative activation of autophagy

JH Teckman et al.

AMERICAN JOURNAL OF PHYSIOLOGY-GASTROINTESTINAL AND LIVER PHYSIOLOGY (2002)

Article Multidisciplinary Sciences

Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector

IH Kim et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2001)

Article Biochemistry & Molecular Biology

Mitochondria recycle Ca2+ to the endoplasmic reticulum and prevent the depletion of neighboring endoplasmic reticulum regions

S Arnaudeau et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2001)