Development of translational read-through-inducing drugs as novel therapeutic options for patients with Fanconi anemia

Development of translational read-through-inducing drugs as novel therapeutic options for patients with Fanconi anemia

Abstract Fanconi anemia (FA) is caused by mutations affecting FANC genes involved in DNA repair, with nearly 20% of FA patients harboring nonsense mutations. Ataluren (PTC124) is a translational read-through-inducing drug (TRID) already approved in Europe that has a well-established safety profile e...

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Main Authors: Anca Manuela Hristodor, Enrico Cappelli, Elena Baldisseri, Roberto Valli, Giuseppe Montalbano, Giovanni Micheloni, Giovanni Porta, Annalisa Frattini, Silvia Ravera, Francesca Fioredda, Giuseppe Lippi, Carlo Dufour, Marco Cipolli, Valentino Bezzerri
Format: Article
Language:English
Published: Nature Publishing Group 2025-06-01
Series:Cell Death Discovery
Online Access:https://doi.org/10.1038/s41420-025-02571-0
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https://doi.org/10.1038/s41420-025-02571-0

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