Mysteries of α1-antitrypsin deficiency: emerging therapeutic strategies for a challenging disease gan Raafe Ghouse, Andrew Chu, Yan Wang, David H. Perlmutter

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A pro-cathepsin L mutant is a luminal substrate for endoplasmic-reticulum-associated degradation in C. elegans. gan Mark T Miedel, Nathan J Graf, Kate E Stephen, Olivia S Long, Stephen C Pak, David H Perlmutter, Gary A Silverman, Cliff J Luke

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

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

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

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A fluorescent reporter for rapid assessment of autophagic flux reveals unique autophagy signatures during C. elegans post-embryonic development and identifies compounds that modula... gan Zachary D. Dawson, Hemalatha Sundaramoorthi, Suk Regmi, Bo Zhang, Stephanie Morrison, Sara M. Fielder, Jessie R. Zhang, Hieu Hoang, David H. Perlmutter, Cliff J. Luke, Gary A. Silverman, Stephen C. Pak

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Resolution of hepatic fibrosis after ZFN-mediated gene editing in the PiZ mouse model of human α1-antitrypsin deficiency gan Yanfeng Li, Chandan Guha, Patrik Asp, Xia Wang, Tatyana L. Tchaikovskya, Kenneth Kim, Matthew Mendel, Gregory J. Cost, David H. Perlmutter, Namita Roy-Chowdhury, Ira J. Fox, Anthony Conway, Jayanta Roy-Chowdhury

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

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Multiple Genes Core to ERAD, Macroautophagy and Lysosomal Degradation Pathways Participate in the Proteostasis Response in α1-Antitrypsin DeficiencySummary gan Jie Li, Francesca Moretti, Tunda Hidvegi, Sanja Sviben, James A.J. Fitzpatrick, Hemalatha Sundaramoorthi, Stephen C. Pak, Gary A. Silverman, Britta Knapp, Ireos Filipuzzi, John Alford, John Reece-Hoyes, Florian Nigsch, Leon O. Murphy, Beat Nyfeler, David H. Perlmutter

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