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DC Field | Value | Language |
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dc.contributor.author | Romani, Luigina | |
dc.contributor.author | Oikonomou, Vasilis | |
dc.contributor.author | Moretti, Silvia | |
dc.contributor.author | Iannitti, Rossana Giulietta | |
dc.contributor.author | D'Adamo, Maria Cristina | |
dc.contributor.author | Villella, Valeria Rachela | |
dc.contributor.author | Pariano, Marilena | |
dc.contributor.author | Sforna, Luigi | |
dc.contributor.author | Borghi, Monica | |
dc.contributor.author | Bellet, Marina M. | |
dc.contributor.author | Fallarino, Francesca | |
dc.contributor.author | Pallotta, Maria Teresa | |
dc.contributor.author | Servillo, Giuseppe | |
dc.contributor.author | Ferrari, Eleonora | |
dc.contributor.author | Puccetti, P. | |
dc.contributor.author | Kroemer, Guido | |
dc.contributor.author | Pessia, Mauro | |
dc.contributor.author | Maiuri, Luigi | |
dc.contributor.author | Goldstein, Allan L. | |
dc.contributor.author | Garaci, Enrico | |
dc.date.accessioned | 2018-03-26T10:19:55Z | |
dc.date.available | 2018-03-26T10:19:55Z | |
dc.date.issued | 2017 | |
dc.identifier.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. | en_GB |
dc.identifier.uri | https://www.um.edu.mt/library/oar//handle/123456789/28254 | |
dc.description.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.) | en_GB |
dc.language.iso | en | en_GB |
dc.publisher | Nature Publishing Group | en_GB |
dc.rights | info:eu-repo/semantics/restrictedAccess | en_GB |
dc.subject | Chloride channels | en_GB |
dc.subject | Cytokines | en_GB |
dc.subject | Immunological adjuvants | en_GB |
dc.subject | Thymosin | en_GB |
dc.title | Thymosin α1 represents a potential potent single-molecule-based therapy for cystic fibrosis | en_GB |
dc.type | article | en_GB |
dc.rights.holder | The copyright of this work belongs to the author(s)/publisher. The rights of this work are as defined by the appropriate Copyright Legislation or as modified by any successive legislation. Users may access this work and can make use of the information contained in accordance with the Copyright Legislation provided that the author must be properly acknowledged. Further distribution or reproduction in any format is prohibited without the prior permission of the copyright holder. | en_GB |
dc.description.reviewed | peer-reviewed | en_GB |
dc.identifier.doi | 10.1038/nm.4305 | |
dc.publication.title | Nature Medicine | en_GB |
Appears in Collections: | Scholarly Works - FacM&SPB |
Files in This Item:
File | Description | Size | Format | |
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Thymosin_α1_represents_a_potential_potent_single_molecule_based_therapy_for_cystic_fibrosis_2017.pdf Restricted Access | 9.67 MB | Adobe PDF | View/Open Request a copy | |
Supplementary_info_Thymosin_α1_represents_a_potential_potent_single-molecule-based_2017.pdf | 2.3 MB | Adobe PDF | View/Open |
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