NEJM· Francesca Ferrua Sabina Cenciarelli Stefania Giannelli Stefania Galimberti Shanmuganathan Chandrakasan Federico Fraschetta Carmen Caputo Davide Sala Ilaria Monti Federica Barzaghi Valeria Calbi Daniele Canarutto Giulia Consiglieri Matteo Doglio Francesca Fumagalli Vera Gallo Maddalena Migliavacca Salvatore Recupero Francesca Tucci Alessia Orsini Raffaella Milani Mariam Datukishvili Simona De Gregori Eugenio Montini Paolo Silvani Matias Soncini Elena Tomasetto Koen van Rossem Laura Castagnaro Federica Miotto Stefano Zancan Celeste Scotti Sean Russell Luigi Naldini Fabio Ciceri Maria Ester Bernardo Suhag Parikh Maria Pia Cicalese and Alessandro Aiutithe WAS Gene Therapy Group*·· 7 天前精选AI 评分62
Etuvetidigene Autotemcel 治疗 Wiskott–Aldrich 综合征的 NEJM 研究
Etuvetidigene Autotemcel for the Treatment of Wiskott–Aldrich Syndrome
AI 导读
NEJM 第 395 卷第 12 期发表 Etuvetidigene Autotemcel 治疗 Wiskott–Aldrich 综合征的研究，页码 1193-1205，时间为 2026 年 9 月 24 日。该文由 Francesca Ferrua 等多位作者及 WAS Gene Therapy Group 完成，来源为 NEJM 当前期目录，feed 仅提供摘要。
推荐理由
NEJM 发表的这项基因治疗研究给出了 Wiskott–Aldrich 综合征患者的疗效与安全性数据，可供了解该罕见病治疗路径。
来源：NEJM · nejm.org