FFC#21/2018

Thymosin alpha 1 in cystic fibrosis: from the lung to the gut

FFC#21/2018

Thymosin alpha 1 in cystic fibrosis: from the lung to the gut

PRINCIPAL INVESTIGATOR

Marina Maria Bellet (Università degli Studi di Perugia, Dip. Medicina Sperimentale)

RESEARCHERS

7

CATEGORY

AREA 4 Lung inflammation

DURATION

1 year

GOAL

€ 40.000 €

RESULTS

Researchers aimed to explore the effect of Tα1 in the gut, using both Tα1 and the pharmaceutical formulation Zadaxin® in two different murine experimental models:1. Model of high fat diet-induced intestinal inflammation, dysbiosis and pancreatic dysfunction, to define the efficacy of Tα1 in a model of “leaky gut”. 2. Model of gastrointestinal infection with C. albicans in CftrF508del mice and Ido1-/- mice. Their results indicated that Tα1 as well Zadaxin are able to ameliorate inflammation and promote barrier function in the gut of CftrF508del, and this effect appears to be IDO1-dependent. In regard to the objective to show the thymosin effect in rescuing CFTR-F508del activity in the same animal models, studies are in progress. The final aim is to repurpose thymosin, a drug in use for different indications, in the field of CF therapies.

OTHER RESULTS

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Two molecules are effective in activating Heat Shock Proteins and enhancing the action of CFTR correctors with the F508del mutation in vitro.

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Some peptide nucleic acids (PNAs) re-sensitise Pseudomonas aeruginosa to the antibiotic meropenem in vitro and reduce its virulence.

FFC#1/2023

Tezacaftor, one of the components of Kaftrio, induces an accumulation of dihydroceramides both in vitro and in vivo in animal models