Target Identification with Live-Cell Photoaffinity Labeling and Mechanism of Action Elucidation of ARN23765, a Highly Potent CFTR Corrector

Authors: Romeo, Elisa, Saccoliti, Francesco, Ocello, Riccardo, Andonaia, Angela, Allegretta, Caterina, Pastorino, Cristina, Pedemonte, Nicoletta, Falchi, Federico, Laselva, Onofrio, Bandiera, Tiziano, Bertozzi, Fabio

Source: Journal of Medicinal Chemistry; February 2025, Vol. 68 Issue: 4 p4596-4618, 23p

Pharmacological rescue of the G85E CFTR variant by preclinical and approved modulators.

Authors: Tomati, Valeria, Capurro, Valeria, Pesce, Emanuela, Pastorino, Cristina, Sondo, Elvira, Lena, Mariateresa, Borrelli, Anna, Cresta, Federico, Pantano, Stefano, Collini, Francesca, Ripani, Pietro, Terlizzi, Vito, Fevola, Cristina, Costa, Stefano, Lucanto, Maria Cristina, Zara, Federico, Bandiera, Tiziano, Bocciardi, Renata, Castellani, Carlo, Galietta, Luis J. V.

Source: Frontiers in Pharmacology; 2024, p1-14, 14p

Subject Terms: CYSTIC fibrosis transmembrane conductance regulator, CYSTIC fibrosis, EPITHELIAL cells, GENETIC disorders, EPITHELIUM

Clinical Consequences and Functional Impact of the Rare S737F CFTR Variant and Its Responsiveness to CFTR Modulators.

Authors: Terlizzi, Vito, Pesce, Emanuela, Capurro, Valeria, Tomati, Valeria, Lena, Mariateresa, Pastorino, Cristina, Bocciardi, Renata, Zara, Federico, Centrone, Claudia, Taccetti, Giovanni, Castellani, Carlo, Pedemonte, Nicoletta

Source: International Journal of Molecular Sciences; Apr2023, Vol. 24 Issue 7, p6576, 15p

Subject Terms: CYSTIC fibrosis transmembrane conductance regulator, CHLORIDE channels, GENETIC profile, MISSENSE mutation, EPITHELIAL cells, CYSTIC fibrosis, SWEAT glands, NASAL mucosa

Gain- and Loss-of-Function CFTR Alleles Are Associated with COVID-19 Clinical Outcomes.

Authors: Baldassarri, Margherita, Zguro, Kristina, Tomati, Valeria, Pastorino, Cristina, Fava, Francesca, Croci, Susanna, Bruttini, Mirella, Picchiotti, Nicola, Furini, Simone, Pedemonte, Nicoletta, Gabbi, Chiara, Renieri, Alessandra, Fallerini, Chiara

Source: Cells (2073-4409); Dec2022, Vol. 11 Issue 24, p4096, 20p

Subject Terms: CYSTIC fibrosis transmembrane conductance regulator, ALLELES, COVID-19

Targeting of Ubiquitin E3 Ligase RNF5 as a Novel Therapeutic Strategy in Neuroectodermal Tumors.

Authors: Principi, Elisa, Sondo, Elvira, Bianchi, Giovanna, Ravera, Silvia, Morini, Martina, Tomati, Valeria, Pastorino, Cristina, Zara, Federico, Bruno, Claudio, Eva, Alessandra, Pedemonte, Nicoletta, Raffaghello, Lizzia

Source: Cancers; Apr2022, Vol. 14 Issue 7, p1802, 21p

Subject Terms: MELANOMA prognosis, IN vitro studies, BIOLOGICAL models, ENERGY metabolism, NEUROBLASTOMA, IN vivo studies, MELANOMA, ENDOPLASMIC reticulum, ANIMAL experimentation, ANTINEOPLASTIC agents, OXIDATIVE stress, CELL survival, GENE expression profiling, NEUROECTODERMAL tumors, CELL lines, MICE, PHARMACODYNAMICS

The L467F-F508del Complex Allele Hampers Pharmacological Rescue of Mutant CFTR by Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Patients: The Value of the Ex Vivo Nasal Epithelial Model to Address Non-Responders to CFTR-Modulating Drugs.

Authors: Sondo, Elvira, Cresta, Federico, Pastorino, Cristina, Tomati, Valeria, Capurro, Valeria, Pesce, Emanuela, Lena, Mariateresa, Iacomino, Michele, Baffico, Ave Maria, Coviello, Domenico, Bandiera, Tiziano, Zara, Federico, Galietta, Luis J. V., Bocciardi, Renata, Castellani, Carlo, Pedemonte, Nicoletta

Source: International Journal of Molecular Sciences; Mar2022, Vol. 23 Issue 6, p3175-3175, 15p

Subject Terms: CHLORIDE channels, CYSTIC fibrosis transmembrane conductance regulator, CYSTIC fibrosis, NASAL mucosa, ALLELES, DRUG therapy

Journey on VX-809-Based Hybrid Derivatives towards Drug-like F508del-CFTR Correctors: From Molecular Modeling to Chemical Synthesis and Biological Assays.

Authors: Parodi, Alice, Righetti, Giada, Pesce, Emanuela, Salis, Annalisa, Tomati, Valeria, Pastorino, Cristina, Tasso, Bruno, Benvenuti, Mirko, Damonte, Gianluca, Pedemonte, Nicoletta, Cichero, Elena, Millo, Enrico

Source: Pharmaceuticals (14248247); Mar2022, Vol. 15 Issue 3, p274, 35p

Subject Terms: BIOSYNTHESIS, BIOLOGICAL assay, CHEMICAL models, CHEMICAL synthesis, SMALL molecules

Partial Rescue of F508del-CFTR Stability and Trafficking Defects by Double Corrector Treatment.

Authors: Capurro, Valeria, Tomati, Valeria, Sondo, Elvira, Renda, Mario, Borrelli, Anna, Pastorino, Cristina, Guidone, Daniela, Venturini, Arianna, Giraudo, Alessandro, Mandrup Bertozzi, Sine, Musante, Ilaria, Bertozzi, Fabio, Bandiera, Tiziano, Zara, Federico, Galietta, Luis J. V., Pedemonte, Nicoletta, Conese, Massimo

Source: International Journal of Molecular Sciences; May2021, Vol. 22 Issue 10, p5262-5262, 1p

Subject Terms: CHLORIDE channels, CYSTIC fibrosis transmembrane conductance regulator, SMALL molecules, PROTEIN folding, CYSTIC fibrosis, UBIQUITINATION

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Rescue by elexacaftor-tezacaftor-ivacaftor of the G1244E cystic fibrosis mutation's stability and gating defects are dependent on cell background.

Authors: Tomati, Valeria¹ (AUTHOR), Costa, Stefano^1,2 (AUTHOR), Capurro, Valeria¹ (AUTHOR), Pesce, Emanuela¹ (AUTHOR), Pastorino, Cristina^1,3 (AUTHOR), Lena, Mariateresa¹ (AUTHOR), Sondo, Elvira¹ (AUTHOR), Di Duca, Marco¹ (AUTHOR), Cresta, Federico⁴ (AUTHOR), Cristadoro, Simona² (AUTHOR), Zara, Federico^1,3 (AUTHOR), Galietta, Luis J.V.^5,6 (AUTHOR), Bocciardi, Renata^1,3 (AUTHOR), Castellani, Carlo⁴ (AUTHOR), Lucanto, Maria Cristina^1,2 (AUTHOR), Pedemonte, Nicoletta¹ (AUTHOR) nicolettapedemonte@gaslini.org

Source: Journal of Cystic Fibrosis. May2023, Vol. 22 Issue 3, p525-537. 13p.

Subject Terms: *CYSTIC fibrosis transmembrane conductance regulator, *CYSTIC fibrosis, *MUTANT proteins, *CELL membranes, *CHLORIDE channels