Capitalizing on the Autophagic Response for Treatment of Liver Disease Caused by Alpha-1-Antitrypsin Deficiency and Other Genetic Diseases

by Andrew S. Chu, David H. Perlmutter, Yan Wang
Published 2014-01-01

Mysteries of α1-antitrypsin deficiency: emerging therapeutic strategies for a challenging disease

by Raafe Ghouse, Andrew Chu, Yan Wang, David H. Perlmutter
Published 2014-04-01

Mechanisms of Action of Autophagy Modulators Dissected by Quantitative Systems Pharmacology Analysis

by Qingya Shi, Fen Pei, Gary A. Silverman, Stephen C. Pak, David H. Perlmutter, Bing Liu, Ivet Bahar
Published 2020-04-01

A pro-cathepsin L mutant is a luminal substrate for endoplasmic-reticulum-associated degradation in C. elegans.

by Mark T Miedel, Nathan J Graf, Kate E Stephen, Olivia S Long, Stephen C Pak, David H Perlmutter, Gary A Silverman, Cliff J Luke
Published 2012-01-01

Deficient and Null Variants of SERPINA1 Are Proteotoxic in a Caenorhabditis elegans Model of α1-Antitrypsin Deficiency.

by Erin E Cummings, Linda P O'Reilly, Dale E King, Richard M Silverman, Mark T Miedel, Cliff J Luke, David H Perlmutter, Gary A Silverman, Stephen C Pak
Published 2015-01-01

Fluphenazine reduces proteotoxicity in C. elegans and mammalian models of alpha-1-antitrypsin deficiency.

by Jie Li, Stephen C Pak, Linda P O'Reilly, Joshua A Benson, Yan Wang, Tunda Hidvegi, Pamela Hale, Christine Dippold, Michael Ewing, Gary A Silverman, David H Perlmutter
Published 2014-01-01

An analog of glibenclamide selectively enhances autophagic degradation of misfolded α1-antitrypsin Z.

by Yan Wang, Murat C Cobanoglu, Jie Li, Tunda Hidvegi, Pamela Hale, Michael Ewing, Andrew S Chu, Zhenwei Gong, Radhika Muzumdar, Stephen C Pak, Gary A Silverman, Ivet Bahar, David H Perlmutter
Published 2019-01-01

Automated high-content live animal drug screening using C. elegans expressing the aggregation prone serpin α1-antitrypsin Z.

by Sager J Gosai, Joon Hyeok Kwak, Cliff J Luke, Olivia S Long, Dale E King, Kevin J Kovatch, Paul A Johnston, Tong Ying Shun, John S Lazo, David H Perlmutter, Gary A Silverman, Stephen C Pak
Published 2010-11-01