FFC#7/2014

A kinase-directed approach to rescue functionality of F508del CFTR

FFC#7/2014

A kinase-directed approach to rescue functionality of F508del CFTR

PRINCIPAL INVESTIGATOR

Andrea Venerando (Dip. Scienze Biomediche, Università di Padova)

Partner

Valeria Rachela Villella (IERFC, Divisione di Genetica e Biologia Cellulare, Istituto San Raffaele, Milano)

RESEARCHERS

13

CATEGORY

AREA 1 Therapies to correct the underlying defect

DURATION

2 years

GOAL

€ 40.000

RESULTS

In vitro and in vivo models of CF were used to demonstrate whether treatment of CF cells and CF mice with potential protein kinase CK2 modulators are able to play a role in the process leading to premature degradation of CFTR-F508del protein. Data were shown indicating that pharmacological inhibition of CK2 prolongs the rescue effect of cysteamine after its whashout. Refining existing leads through a target-driven drug discovery approach may be useful to identify old/new and more efficacious compounds which favour a better and larger rescue of mutant CFTR.

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.

FFC#5/2024

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