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MARCO TUTONE

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

  • Autori: Lentini, L; Pibiri, I; Melfi, R; Tutone, M; Pace, A; Barone, G; Costantino, C; Di Leonardo, A
  • Anno di pubblicazione: 2014
  • Tipologia: eedings
  • OA Link: http://hdl.handle.net/10447/102071

Abstract

Cystic Fibrosis patients with nonsense-mutation in h-CFTR gene generally make virtually no CFTR protein and thus often have a more severe form of CF. Ataluren (PTC124) was suggested to induce read-through of premature but not normal termination codons. Despite the promising results there is not a general consensus on the mechanism of its action (protein stabilization or codon read-through) and its efficacy, the identification of new PTC124 analogues and the study of the mechanism of action may led to a new strategy for the development of a pharmacologic approach to the cure of CF.