Enhancing autophagy with drugs or lung-directed gene therapy reverses the pathological effects of respiratory epithelial cell proteinopathy

Tunda Hidvegi, Donna B. Stolz, John F. Alcorn, Samuel A. Yousem, Jieru Wang, Adriana S. Leme, A. Mc Garry Houghton, Pamela Hale, Michael Ewing, Houming Cai, Evelyn Akpadock Garchar, Nunzia Pastore, Patrizia Annunziata, Naftali Kaminski, Joseph Pilewski, Steven D. Shapiro, Stephen C. Pak, Gary A. Silverman, Nicola Brunetti-Pierri, David H. Perlmutter

Research output: Contribution to journalArticlepeer-review

21 Scopus citations

Abstract

Background: Several rare lung proteinopathies are characterized by lung fibrosis due to accumulation of misfolded proteins in epithelial cells. Results: The PiZ mouse models the pathological characteristics of these lung proteinopathies, and the pathology is ameliorated by autophagy enhancing therapies. Conclusion: Autophagy represents a key proteostasis mechanism for lung proteinopathies and a potential therapeutic target. Significance: The PiZ mouse is an attractive animal model of lung proteinopathies.

Original languageEnglish
Pages (from-to)29742-29757
Number of pages16
JournalJournal of Biological Chemistry
Volume290
Issue number50
DOIs
StatePublished - Dec 11 2015
Externally publishedYes

Fingerprint Dive into the research topics of 'Enhancing autophagy with drugs or lung-directed gene therapy reverses the pathological effects of respiratory epithelial cell proteinopathy'. Together they form a unique fingerprint.

Cite this