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Background. Cystic Fibrosis patients with nonsense-mutation in the CFTR gene generally make virtually no CFTR protein and thus often have a more severe form of CF. Recently, Ataluren (formerly PTC124) was suggested to induce the read-through of premature termination codons (PTCs) mainly the UGA codon. However, despite promising results there is not a general consensus of Ataluren efficacy and mechanism of action.

postdoc journal

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