| 2026 |
Delivering the message: mRNA replacement reaches the liver in Niemann-Pick type C1 disease |
Nota o commento |
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| 2026 |
Beyond the stop: Oxadiazole TRIDs restore LRBA protein expression in nonsense-driven primary immunodeficiency |
Articolo in rivista |
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| 2025 |
CFTR rescue in a G542X nonsense mouse model of cystic fibrosis: investigating the mechanism of action and activity of translational readthrough-inducing drugs |
Abstract in rivista |
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| 2025 |
Nonsense Mutations in Rare and Ultra-Rare Human Disorders: An Overview |
Articolo in rivista |
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| 2025 |
PERSONALIZED MEDICINE FIGHTING NONSENSE DISEASES: EXPLORATION OF NV MOLECULES MECHANISM OF ACTION IN PURE-LITE SYSTEM AND THEIR READTHROUGH ACTIVITY IN CHOROIDEREMIA NONSENSE MODEL |
Abstract in rivista |
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| 2025 |
A new approach against nonsense mutations: Translational Readthrough Inducing Drugs to induce rescue of p53 |
Poster pubblicato in volume |
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| 2025 |
CFTR rescue in W1282X Cystic fibrosis patients-derived intestinal organoids (PDIOs) mediated by translational readthrough-inducing drugs (TRIDs) |
Articolo in rivista |
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| 2025 |
Rescuing nonsense in cancer: recovering p53 tumor suppressor gene expression by
translational readthrough inducing drugs (TRIDs) |
Poster pubblicato in volume |
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| 2024 |
Promoting readthrough of nonsense mutations in CF mouse model: Biodistribution and efficacy of NV848 in rescuing CFTR protein expression |
Articolo in rivista |
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| 2024 |
A precision medicine approach for nonsense mutations: study of novel small molecules for the rescue of p53 protein expression |
Abstract non pubblicato |
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| 2023 |
Readthrough Approach Using NV Translational Readthrough-Inducing Drugs (TRIDs): A Study of the Possible Off-Target Effects on Natural Termination Codons (NTCs) on TP53 and Housekeeping Gene Expression |
Articolo in rivista |
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| 2023 |
Production of CHM R293X cell model system to study the rescue of the Rab Escort Protein-1 expression by TRIDs nonsense suppression activity |
Abstract in atti di convegno pubblicato in volume |
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| 2022 |
Readthrough Inducing Drugs (TRIDs) in human fibroblasts harboring the c.5047 C>T (R1683*) nonsense mutation |
Abstract in atti di convegno pubblicato in volume |
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