Beyond the stop: Oxadiazole TRIDs restore LRBA protein expression in nonsense-driven primary immunodeficiency
超越终点:恶二唑类TRIDs可恢复无义突变驱动的原发性免疫缺陷中的LRBA蛋白表达
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.1
doi:10.1016/j.omtn.2025.102808
Fiduccia, Ignazio; Vitale, Emanuele; Varrica, Riccardo; Ricci, Davide; Marino, Sefora; Zito, Antonino; Pace, Andrea; Colige, Alain; Moutschen, Michel; Borutzki, Yasmin; Bileck, Andrea; Meier-Menches, Samuel M; Lentini, Laura; Pibiri, Ivana