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

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Title: Development of translational read-through-inducing drugs as novel therapeutic options for patients with Fanconi anemia.
Authors: Hristodor AM; Cystic Fibrosis Center, Azienda Ospedaliera Universitaria Integrata, Verona, Italy., Cappelli E; Unit of Hematology, IRCCS G. Gaslini, Genoa, Italy., Baldisseri E; Cystic Fibrosis Center, Azienda Ospedaliera Universitaria Integrata, Verona, Italy., Valli R; Department of Medicine and Surgery, University of Insubria, Varese, Italy.; Genomic Medicine Center, University of Insubria, Varese, Italy., Montalbano G; Department of Medicine and Surgery, University of Insubria, Varese, Italy.; Genomic Medicine Center, University of Insubria, Varese, Italy., Micheloni G; Department of Medicine and Surgery, University of Insubria, Varese, Italy.; Genomic Medicine Center, University of Insubria, Varese, Italy., Porta G; Department of Medicine and Surgery, University of Insubria, Varese, Italy.; Genomic Medicine Center, University of Insubria, Varese, Italy., Frattini A; Institute for Genetic and Biomedical Research (IRGB), Consiglio Nazionale delle Ricerche, Milan, Italy., Ravera S; Department of Experimental Medicine, University of Genoa, Genoa, Italy., Fioredda F; Unit of Hematology, IRCCS G. Gaslini, Genoa, Italy., Lippi G; Unit of Clinical Biochemistry, Department of Engineering for Innovation Medicine, University of Verona, Verona, Italy., Dufour C; Unit of Hematology, IRCCS G. Gaslini, Genoa, Italy., Cipolli M; Cystic Fibrosis Center, Azienda Ospedaliera Universitaria Integrata, Verona, Italy., Bezzerri V; Cystic Fibrosis Center, Azienda Ospedaliera Universitaria Integrata, Verona, Italy. v.bezzerri@unilink.it.; Department of Life Sciences, Health and Health Professions, Link Campus University, Rome, Italy. v.bezzerri@unilink.it.
Source: Cell death discovery [Cell Death Discov] 2025 Jun 21; Vol. 11 (1), pp. 286. Date of Electronic Publication: 2025 Jun 21.
Publication Type: Journal Article
Journal Info: Publisher: Nature Publishing Group Country of Publication: United States NLM ID: 101665035 Publication Model: Electronic Cited Medium: Print ISSN: 2058-7716 (Print) Linking ISSN: 20587716 NLM ISO Abbreviation: Cell Death Discov Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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ISSN:2058-7716
DOI:10.1038/s41420-025-02571-0