FFC#01/2014

Identification and validation of novel molecules obtained by integrated computational and experimental approaches for the read-through of PTCs in CF cells

FFC#01/2014

Identification and validation of novel molecules obtained by integrated computational and experimental approaches for the read-through of PTCs in CF cells

PRINCIPAL INVESTIGATOR

Laura Lentini (Dip. di Biologia, Scienze Chimiche e Farmaceutiche e Tecnologie -STEBICEF, Sez. di Biologia Cellulare, Università di Palermo)

Partner

Ivana Pibiri (Dip. di Biologia, Scienze Chimiche e Farmaceutiche e Tecnologie – STEBICEF, Sez. di Biologia Cellulare, Università di Palermo)

RESEARCHERS

7

CATEGORY

AREA 1 Therapies to correct the underlying defect

DURATION

2 years

GOAL

€ 38.000 €

RESULTS

Eighteen analogues of Ataluren (PTC124) were synthesized and tested in three different biological models. Ten of these new compounds showed high read-through capacity. New insight about their mechanism of action was obtained.

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