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

Beyond readthrough: ataluren restores mitochondrial function and reduces oxidative stress in FANCA-mutated cells via mTOR-DRP1 modulation.

超越读通:ataluren 通过 mTOR-DRP1 调节恢复 FANCA 突变细胞的线粒体功能并降低氧化应激。

Balbi Matilde, Guidi Elisa, Hristodor Anca Manuela, Corsolini Fabio, Cossu Vanessa, Bottega Roberta, Serra Martina, Pestarino Sara, Bartolucci Martina, Cipolli Marco, Regis Stefano, Bezzerri Valentino, Cappelli Enrico, Ravera Silvia

Modulation of immune responses by elexacaftor/tezacaftor/ivacaftor therapy in cystic fibrosis: data from a compassionate use program

elexacaftor/tezacaftor/ivacaftor 疗法对囊性纤维化患者免疫反应的调节:来自同情用药计划的数据

Lucca, Francesca; Pianazzola, Anna; Meneghelli, Ilaria; Catanzaro, Giuseppina; Sabbioni, Giuseppe; Tridello, Gloria; Hristodor, Anca Manuela; De Luca, Erik; Menichetti, Giacomo; Breveglieri, Giulia; Altieri, Maria Teresa; Bordicchia, Marica; Fabrizzi, Benedetta; Ferretti, Elisabetta; Lippi, Giuseppe; Ros, Mirco; Borgatti, Monica; Cipolli, Marco; Bezzerri, Valentino

Ataluren improves hematopoietic and pancreatic disorders in Shwachman-Diamond syndrome patients: a compassionate program case-series.

Ataluren 改善了施瓦赫曼-戴蒙德综合征患者的造血和胰腺疾病:一项关怀计划病例系列

Bezzerri Valentino, Pegoraro Anna, Hristodor Anca Manuela, Crane Genevieve M, Meneghelli Ilaria, Brignole Cecilia, Boni Christian, Baldisseri Elena, Vella Antonio, Menichetti Giacomo, Valli Roberto, Porta Giovanni, D'Amico Giovanna, Tecchio Cristina, Parisi Alice, Lippi Giuseppe, Mercuri Angela, Cesaro Simone, Corey Seth J, Cipolli Marco

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

开发转化阅读通读诱导药物作为范可尼贫血患者的新型治疗选择

Hristodor Anca Manuela, Cappelli Enrico, Baldisseri Elena, Valli Roberto, Montalbano Giuseppe, Micheloni Giovanni, Porta Giovanni, Frattini Annalisa, Ravera Silvia, Fioredda Francesca, Lippi Giuseppe, Dufour Carlo, Cipolli Marco, Bezzerri Valentino

CFTR modulators response of S737F and T465N CFTR variants on patient-derived rectal organoids

CFTR 调节剂对患者来源的直肠类器官中 S737F 和 T465N CFTR 变体的反应

Karina Kleinfelder, Paola Melotti, Anca Manuela Hristodor, Cristina Fevola, Giovanni Taccetti, Vito Terlizzi, Claudio Sorio

Theratyping of the Rare CFTR Genotype A559T in Rectal Organoids and Nasal Cells Reveals a Relevant Response to Elexacaftor (VX-445) and Tezacaftor (VX-661) Combination

直肠类器官和鼻腔细胞中罕见 CFTR 基因型 A559T 的治疗分型揭示了对 Elexacaftor (VX-445) 和 Tezacaftor (VX-661) 组合的相关反应

Karina Kleinfelder, Valeria Rachela Villella, Anca Manuela Hristodor, Carlo Laudanna, Giuseppe Castaldo, Felice Amato, Paola Melotti, Claudio Sorio