Development of a small molecule that corrects misfolding and increases secretion of Z α1-antitrypsin
D. Lomas, J. A. Irving, C. Arico-Muendel, S. Belyanskaya, A. Brewster, M. J. B. Brown, C.-w. Chung, H. Dave, A. Denis, N. Dodic, A. Dossang, P. Eddershaw, D. Klimaszewska, I. Haq, D. Holmes, , A. C. Pearce
This study identifies a small molecule that stabilizes Z α1-antitrypsin, reduces polymerization, and increases its secretion in cells and mice.
bioRxiv · Jul 26, 2020Pharmacology & Toxicology