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*·· 13 天前AI 评分20
Etuvetidigene Autotemcel 治疗 Wiskott–Aldrich 综合征
Etuvetidigene Autotemcel for the Treatment of Wiskott–Aldrich Syndrome
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Etuvetidigene Autotemcel（一种自体造血干细胞基因疗法）在 Wiskott–Aldrich 综合征患者中显示出疗效，研究发表于 NEJM 第 395 卷第 12 期。该疗法通过基因修正自体干细胞恢复免疫和血小板功能，为这一罕见原发性免疫缺陷病提供了潜在的一次性治疗方案。具体临床终点数据需查阅原文。
来源：NEJM · nejm.org