After virtual screening, we synthesized a small library of analogues of the NV2445 molecule and tested them by FLuc assay and in different biological models. These new compounds showed high read-through capacity and CFTR rescue. Moreover, in vitro and in vivo toxicity test (Zebrafish) was performed to assess the safety profile of the molecules synthesized: one of them resulted without toxicity.

Congress abstracts

• Lentini, Melfi R, Tutone M et al. Identification of a new molecule with readthrough activity to rescue CFTR protein function, 5° meeting biotecnologie, ricerca di base, interdisciplinare, traslazionale in ambito biomedico, Palermo, 5-6 luglio 2018
• Lentini L, Pibiri I, Melfi R. Rescue of nonsense mutations by novel readthrough molecules in biological model systems and in cystic fibrosis cells, Convegno AGI, 7-9 Settembre 2017, Cortona
• Pibiri, Lentini L, Tutone M et al. Rescuing CFTR Protein Function: 1,3,4-oxadiazoles versus 1,2,4-oxadiazoles as readthrough inducing drugs, Italian-Spanish-Portuguese joint Meeting in Medicinal Chemistry MedChemSicily, Palermo 17-18 Luglio 2018
• Lentini L. Rescuing mutant CFTR in cystic fibrosis by genetic drugs, XIII Summer School on Advanced Biotechnology, Orto Botanico, Palermo, 2-5 Settembre 2018
• Campofelice A, Culletta G, Tutone M et al. Uno studio comparativo in silico sui possibili target di Ataluren e analoghi farmaci promotori di readthrough di codoni di stop prematuri, Convegno congiunto delle Sezioni di Calabria e Sicilia 2019. Palermo, 1-2 marzo 2019
• Pibiri I, Pace A, Tutone M et al. Derivati ossadiozolici per il trattamento della fibrosi cistica: readthrough di mutazioni nonsense, Convegno congiunto delle Sezioni di Calabria e Sicilia 2019. Palermo, 1-2 marzo 2019
• Campofelice A, Ricco Galluzzo P, Lentini L et al. New molecules as translational readthrough promoters of nonsense mutations: rescuing the CFTR protein, XXXIX Convegno nazionale della Divisione di Chimica Organica della Società Chimica Italiana, Torino, 8-12 settembre 2019
• Campofelice A, Ricco Galluzzo P, Lentini L et al. New molecules as translational readthrough promoters of nonsense mutations: rescuing the CFTR protein, XXXIX Convegno Nazionale della Divisione di Chimica Organica, CDCO, 8-12 settembre 2019, Torino