Please use this identifier to cite or link to this item:
Title: Thymosin α1 represents a potential potent single-molecule-based therapy for cystic fibrosis
Authors: Romani, Luigina
Oikonomou, Vasilis
Moretti, Silvia
Iannitti, Rossana Giulietta
D'Adamo, Maria Cristina
Villella, Valeria Rachela
Pariano, Marilena
Sforna, Luigi
Borghi, Monica
Bellet, Marina M.
Fallarino, Francesca
Pallotta, Maria Teresa
Servillo, Giuseppe
Ferrari, Eleonora
Puccetti, P.
Kroemer, Guido
Pessia, Mauro
Maiuri, Luigi
Goldstein, Allan L.
Garaci, Enrico
Keywords: Chloride channels
Immunological adjuvants
Issue Date: 2017
Publisher: Nature Publishing Group
Citation: Romani, L., Oikonomou, V., Moretti, S., Iannitti, R. G., D'Adamo, M. C., Villella, V. R.,...Garaci, E. (2017). Thymosin α1 represents a potential potent single-molecule-based therapy for cystic fibrosis. Nature Medicine, 23(5), 590-600.
Abstract: Cystic fibrosis (CF) is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) that compromise its chloride channel activity. The most common mutation, p.Phe508del, results in the production of a misfolded CFTR protein, which has residual channel activity but is prematurely degraded. Because of the inherent complexity of the pathogenetic mechanisms involved in CF, which include impaired chloride permeability and persistent lung inflammation, a multidrug approach is required for efficacious CF therapy. To date, no individual drug with pleiotropic beneficial effects is available for CF. Here we report on the ability of thymosin alpha 1 (Tα1) - a naturally occurring polypeptide with an excellent safety profile in the clinic when used as an adjuvant or an immunotherapeutic agent - to rectify the multiple tissue defects in mice with CF as well as in cells from subjects with the p.Phe508del mutation. Tα1 displayed two combined properties that favorably opposed CF symptomatology: it reduced inflammation and increased CFTR maturation, stability and activity. By virtue of this two-pronged action, Tα1 has strong potential to be an efficacious single-molecule-based therapeutic agent for CF. (Supplementary information accompanies this paper in the files section.)
Appears in Collections:Scholarly Works - FacM&SPB

Items in OAR@UM are protected by copyright, with all rights reserved, unless otherwise indicated.