日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

A precision medicine approach to primary immunodeficiency disease: Ataluren strikes nonsense mutations once again.

针对原发性免疫缺陷疾病的精准医疗方法:阿塔卢伦再次击中无义突变

Lentini Laura, Perriera Riccardo, Corrao Federica, Melfi Raffaella, Tutone Marco, Carollo Pietro S, Fiduccia Ignazio, Pace Andrea, Ricci Davide, Genovese Francesco, Colige Alain, Delvenne Philippe, Grimbacher Bodo, Moutschen Michel, Pibiri Ivana

Promoting readthrough of nonsense mutations in CF mouse model: Biodistribution and efficacy of NV848 in rescuing CFTR protein expression

促进囊性纤维化小鼠模型中无义突变的通读:NV848在挽救CFTR蛋白表达中的生物分布和功效

Fiduccia, Ignazio; Corrao, Federica; Zizzo, Maria Grazia; Perriera, Riccardo; Genovese, Francesco; Vitale, Emanuele; Ricci, Davide; Melfi, Raffaella; Tutone, Marco; Pace, Andrea; Lentini, Laura; Pibiri, Ivana

Investigating the Inhibition of FTSJ1, a Tryptophan tRNA-Specific 2'-O-Methyltransferase by NV TRIDs, as a Mechanism of Readthrough in Nonsense Mutated CFTR

研究NV TRIDs对色氨酸tRNA特异性2'-O-甲基转移酶FTSJ1的抑制作用,作为无义突变CFTR中通读的机制

Carollo, Pietro Salvatore; Tutone, Marco; Culletta, Giulia; Fiduccia, Ignazio; Corrao, Federica; Pibiri, Ivana; Di Leonardo, Aldo; Zizzo, Maria Grazia; Melfi, Raffaella; Pace, Andrea; Almerico, Anna Maria; Lentini, Laura

Site-Specific RNA Editing of Stop Mutations in the CFTR mRNA of Human Bronchial Cultured Cells

人支气管培养细胞中CFTR mRNA终止突变的位点特异性RNA编辑

Chiavetta, Roberta F; Titoli, Simona; Barra, Viviana; Cancemi, Patrizia; Melfi, Raffaella; Di Leonardo, Aldo

Strategies against Nonsense: Oxadiazoles as Translational Readthrough-Inducing Drugs (TRIDs)

对抗无义突变的策略:恶二唑类化合物作为翻译通读诱导药物(TRIDs)

Campofelice, Ambra; Lentini, Laura; Di Leonardo, Aldo; Melfi, Raffaella; Tutone, Marco; Pace, Andrea; Pibiri, Ivana

Caffeine boosts Ataluren's readthrough activity

咖啡因能增强阿塔鲁伦的阅读活性

Lentini, Laura; Melfi, Raffaella; Cancemi, Patrizia; Pibiri, Ivana; Di Leonardo, Aldo