Alpha-1 Study Additional Publications

A Preliminary Study of the Efficacy and Safety of Carbamazepine in Severe Liver Disease Due to Alpha-1 Antitrypsin Deficiency

Published Articles

Below is a partial list of published papers related to this research study.

A C. elegans model of human α1-antitrypsin deficiency links RNAi to misfolded protein turnover
OS Long, JA Benson, JH Kwak, CJ Luke, SJ Gosai, LP O'Reilly, Y Wang, J Li, AC Vetica, MT Miedel, DB Stolz, SC Watkins, S Züchner, DH Perlmutter, GA Silverman, SC Pak
Human Molecular Genetics
2014 Oct 16

A genome-wide RNAi screen identifies potential drug targets in a C. elegans model of α1-antitrypsin deficiency
LP O'Reilly, OS. Long, MC Cobanoglu, JA Benson, CJ Luke, MT Miedel, P Hale, DH Perlmutter, I Bahar, GA Silverman, SC Pak
Human Molecular Genetics
2014 Oct 16

C. elegans in high-throughput drug discovery
LP O'Reilly, CJ Luke, DH Perlmutter, GA Silverman, SC Pak
Advanced Drug Delivery Reviews
2014 April

Fluphenazine reduces protetotoxicity in C. elegans and mammalian models of alpha-1-antitrypsin deficiency (PDF)
J Li, SC Pak, LP O’Reilly, JA Benson, Y Wang, T Hidvegi, P Hale, C Dippold, M Ewing, GA Silverman, DH Perlmutter
PLOS ONE
2014 Jan 31

Mysteries of α1-antitrypsin deficiency: Emerging therapeutic strategies for a challenging disease
R Ghouse, AS Chu, Y Wang, DH Perlmutter
Disease Models & Mechanisms
2014

Capitalizing on the autophagic response for treatment of liver disease caused by alpha-1-antitrypsin deficiency and other genetic diseases
AS Chu, DH Perlmutter, Y Wang
BioMed Research International
2014

Worming our way to novel drug discovery with the Caenorhabditis elegans proteostasis network, stress response and insulin-signaling pathways
LP O'Reilly, JA Benson, EE Cummings, DH Perlmutter, GA Silverman, SC Pak
Expert Opinion Drug Discoveries
2014

Targeting intracellular degradation pathways for treatment of childhood liver disease due to α1-antitrypsin deficiency (PDF)
Y Wang, DH Perlmutter
Pediatric Research
2013 Dec 11

Automated high-content live animal drug screening using C. elegans expressing the aggregation prone serpin α1-antitrypsin (PDF)
SJ Gosai, JH Kwak, CJ Luke, OS Long, DE King, KJ Kovatch, PA Johnston, TY Shun, JS Lazo, DH Perlmutter, GA Silverman, SC Pak
PLOS ONE
2010 Nov 12

An autophagy-enhancing drug promotes degradation of mutant α-antitrypsin Z and reduces hepatic fibrosis
T Hidvegi, M Ewing, P Hale, C Dippold, C Beckett, C Kemp, N Maurice, A Mukherjee, C Goldbach, S Watkins, G Michalopoulos, DH Perlmutter
Science
2010 Jul 9