Adenine base editing with engineered virus-like particles rescues the CFTR mutation G542X in patient-derived intestinal organoids
利用工程改造的病毒样颗粒进行腺嘌呤碱基编辑,可挽救患者来源的肠道类器官中的 CFTR 突变 G542X。
期刊:iScience
影响因子:4.6
doi:10.1016/j.isci.2025.111979
Lucia Nicosia,Iwona Pranke,Roberta V Latorre,Joss B Murray,Lisa Lonetti,Kader Cavusoglu-Doran,Elise Dreano,James P Costello,Michael Carroll,Paola Melotti,Claudio Sorio,Isabelle Sermet-Gaudelus,Martina F Scallan,Patrick T Harrison