Thymosin α1 represents a potential potent single-molecule-based therapy for cystic fibrosis

• Published in: Nature Medicine Vol. 23; no. 5; pp. 590 - 600
• Main Authors: Romani, Luigina, Oikonomou, Vasilis, Moretti, Silvia, Iannitti, Rossana G, D'Adamo, Maria Cristina, Villella, Valeria R, Pariano, Marilena, Sforna, Luigi, Borghi, Monica, Bellet, Marina M, Fallarino, Francesca, Pallotta, Maria Teresa, Servillo, Giuseppe, Ferrari, Eleonora, Puccetti, Paolo, Kroemer, Guido, Pessia, Mauro, Maiuri, Luigi, Goldstein, Allan L, Garaci, Enrico
• Format: Journal Article Magazine Article
• Language: English
• Published: New York Nature Publishing Group US 01.05.2017; Nature Publishing Group
• Subjects: 13/1; 13/109; 13/21; 13/89; 14/63; 38/61; 64/60; 692/308; 692/308/1426; 82/80; 9/74; Adjuvants, Immunologic - pharmacology; Animals; Autophagy - drug effects; Biomedicine; Blotting, Western; Cancer Research; Cell Line; Chloride Channels - drug effects; Chloride Channels - metabolism; Cystic Fibrosis - genetics; Cystic Fibrosis - immunology; Cystic Fibrosis - metabolism; Cystic Fibrosis Transmembrane Conductance Regulator - drug effects; Cystic Fibrosis Transmembrane Conductance Regulator - genetics; Cystic Fibrosis Transmembrane Conductance Regulator - metabolism; Cytokines - drug effects; Cytokines - immunology; Disease Models, Animal; Epithelial Cells - drug effects; Epithelial Cells - metabolism; Fluorescent Antibody Technique; Humans; Immunohistochemistry; Immunoprecipitation; Indoleamine-Pyrrole 2,3,-Dioxygenase - drug effects; Indoleamine-Pyrrole 2,3,-Dioxygenase - immunology; Infectious Diseases; Inflammation; Life Sciences; Metabolic Diseases; Mice; Mice, Inbred CFTR; Molecular Medicine; Neurosciences; Patch-Clamp Techniques; Protein Stability - drug effects; RAW 264.7 Cells; Respiratory Mucosa - cytology; Thymalfasin; Thymosin - analogs & derivatives; Thymosin - pharmacology; Ubiquitin Thiolesterase - drug effects; Ubiquitin Thiolesterase - metabolism; Ubiquitination - drug effects

Thymosin α1 is used in the clinic as a treatment in viral disease and acts as an anti-inflammatory. Here it was found to also correct the misfolding of mutant CTFR and potentiate its activity, thus improving outcome in a mouse model of cystic fibrosis. Cystic fibrosis (CF) is caused by mutations in...