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FFC#03/2015

Assessment and pharmacological correction of abnormalities in bicarbonate (HCO3-) and mucus transport in intestinal biopsies and organoids of CF patients

FFC#03/2015

Assessment and pharmacological correction of abnormalities in bicarbonate (HCO3-) and mucus transport in intestinal biopsies and organoids of CF patients

PRINCIPAL INVESTIGATOR

Hugo de Jonge (Dipartimento di Gastroenterologia ed Epatologia – Centro Medico, Erasmus University, Rotterdam)

Partner

Sara Caldrer (Dip. di Patologia e Diagnostica, sezione di Patologia Generale – Università di Verona)

RESEARCHERS

11

CATEGORY

AREA 1 Therapies to correct the underlying defect

DURATION

1 year

GOAL

€ 45.000 €

RESULTS

The bicarbonate secretion resulted absent in F508del organoids. The corrector VX-809 and potentiator VX-770 rescued bicarbonate secretion in F508del organoids more efficient than chloride secretion. The protocols developed may facilitate future preclinical testing of novel CFTR repair molecules for their ability to restore bicarbonate transport in organoids from individual CF patients.

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OTHER RESULTS

FFC #3/2024

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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FFC#5/2024

Some peptide nucleic acids (PNAs) re-sensitise Pseudomonas aeruginosa to the antibiotic meropenem in vitro and reduce its virulence.
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FFC#1/2023

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