FFC#08/2015

Dissecting the role of TG2 in cystic fibrosis pathogenesis: identification of possible novel therapeutic targets

FFC#08/2015

Dissecting the role of TG2 in cystic fibrosis pathogenesis: identification of possible novel therapeutic targets

PRINCIPAL INVESTIGATOR

Mauro Piacentini (Dipartimento di Biologia – Università di Tor Vergata, Roma)

Partner

Luigi Maiuri (IERFC, Istituto Europeo per la Ricerca in Fibrosi Cistica – Istituto Scientifico San Raffaele, Milano)

RESEARCHERS

13

CATEGORY

AREA 1 Therapies to correct the underlying defect

DURATION

2 years

GOAL

€ 40.000 €

RESULTS

The researchers shew that ablation of TG2 function in vivo improved the disease phenotype of CFTR-F508del mice that exhibited increased channel functionality, improved intestinal function, reduced inflammation, as well as resistance to infection with Pseudomonas aeruginosa. TG2, by its protein disulphide isomerase activity, triggers the trimerization and activation of HSF1, the master transcription factor that regulates the response to proteotoxic stress, promoting the induction of stress-inducibleible chaperone HSP70. CF mice as well as primary nasal epithelial cells derived from CF patients usually displayed increased levels of HSF1 and HSP70 expression as compared to normal controls. The researchers found that these alterations were significantly ameliorated by genetic TG2 ablation as well as by its pharmacological inhibition with cysteamine, which indeed suppressed the protein disulfide-isomerase activity of TG2.The perspective is to identify protective agents that modulating TG2 activity could restore CFTR proteostasis.

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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