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

Guidelines for the use and interpretation of assays for monitoring autophagy

(2012) Daniel J. Klionsky et al.   Autophagy

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

(2012) Carmine Settembre et al.   EMBO JOURNAL

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

(2012) Sam Alam et al.   JOURNAL OF HEPATOLOGY

Balloon Catheter Delivery of Helper-dependent Adenoviral Vector Results in Sustained, Therapeutic hFIX Expression in Rhesus Macaques

(2012) Nicola Brunetti-Pierri et al.   MOLECULAR THERAPY

Sustained miRNA-mediated Knockdown of Mutant AAT With Simultaneous Augmentation of Wild-type AAT Has Minimal Effect on Global Liver miRNA Profiles

(2012) Christian Mueller et al.   MOLECULAR THERAPY

Transcriptional Activation of Lysosomal Exocytosis Promotes Cellular Clearance

(2011) Diego L. Medina et al.   DEVELOPMENTAL CELL

Helper-dependent adenoviral vectors for liver-directed gene therapy

(2011) N. Brunetti-Pierri et al.   HUMAN MOLECULAR GENETICS

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

(2011) P. J. Khandelwal et al.   HUMAN MOLECULAR GENETICS

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

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

TFEB Links Autophagy to Lysosomal Biogenesis

(2011) C. Settembre et al.   SCIENCE

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

(2010) Shalesh Kaushal et al.   EXPERIMENTAL BIOLOGY AND MEDICINE

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

(2010) Kelly Jean Thomas et al.   HUMAN MOLECULAR GENETICS

Chromosomal Integration of Adenoviral Vector DNA In Vivo

(2010) S. L. Stephen et al.   JOURNAL OF VIROLOGY

An Autophagy-Enhancing Drug Promotes Degradation of Mutant  1-Antitrypsin Z and Reduces Hepatic Fibrosis

(2010) T. Hidvegi et al.   SCIENCE

Reduction of Protein Translation and Activation of Autophagy Protect against PINK1 Pathogenesis in Drosophila melanogaster

(2010) Song Liu et al.   PLoS Genetics

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

(2009) Nicola Brunetti-Pierri et al.   CURRENT GENE THERAPY

Bioengineered Factor IX Molecules with Increased Catalytic Activity Improve the Therapeutic Index of Gene Therapy Vectors for Hemophilia B

(2009) Nicola Brunetti-Pierri et al.   HUMAN GENE THERAPY

Host Cell Detection of Noncoding Stuffer DNA Contained in Helper-Dependent Adenovirus Vectors Leads to Epigenetic Repression of Transgene Expression

(2009) P. J. Ross et al.   JOURNAL OF VIROLOGY

Lysosome biogenesis and lysosomal membrane proteins: trafficking meets function

(2009) Paul Saftig et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

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

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Autophagic disposal of the aggregation-prone protein that causes liver inflammation and carcinogenesis in α-1-antitrypsin deficiency

(2008) D H Perlmutter   CELL DEATH AND DIFFERENTIATION

Efficient, Long-term Hepatic Gene Transfer Using Clinically Relevant HDAd Doses by Balloon Occlusion Catheter Delivery in Nonhuman Primates

(2008) Nicola Brunetti-Pierri et al.   MOLECULAR THERAPY