FFC#6/2014

Development of novel methodologies for the identification of CFTR-targeted drugs: a multidisciplinary approach using Real Time Surface Plasmon Resonance interaction assay supported by bioinformatics strategies on HPC infrastructures

FFC#6/2014

Development of novel methodologies for the identification of CFTR-targeted drugs: a multidisciplinary approach using Real Time Surface Plasmon Resonance interaction assay supported by bioinformatics strategies on HPC infrastructures

PRINCIPAL INVESTIGATOR

Marco Rusnati (Dip. di Medicina Molecolare e Traslazionale, Università di Brescia, Unità Analisi Interazione Macromolecolare-Sez. di Oncologia Sperimentale e Immunologia)

Partner

Paola Fossa (Dip. di Farmacia, Genova) Alessandro Orro (Istituto di Tecnologie Biomediche, CNR, Segrate-Milano)

RESEARCHERS

11

CATEGORY

AREA 1 Therapies to correct the underlying defect

DURATION

1 year

GOAL

€ 38.000

RESULTS

Molecular dynamics unmasked two binding pockets (BPs) in NBD1. BP1 is smaller in wild type (WT) than in DF508-CFTR, due to two loops (drug site 1 and 2) making contact and closing the BPs. In DF508-NBD1, all the ligands tested bind BP1, VX809 and FCG in drug site 1, EN371B, EN277I and EN371A in drug site 2. VX809 binding to DF508- NBD1 is stable and samples the same space of the WT, indicating a conformational recovery. FCG association is unstable, indicating a different binding site. EN371A is inactive. SPR validated molecular modelling. When the compounds were assayed for their capacity to affect the DF508-NBD1/K8 interaction, EN277I was ineffective while EN371B prevented the interaction, indicating a competition with K8 for the same BP. SPR analysis validates and complements in silico predictions sustaining the integration of bioinformatics and SPR as effective in speeding up the discovery of new CFTR-targeted drugs.

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