Integrare Research Unit UMR_S951 , Genethon, Inserm, Univ Evry, EPHE, Evry, France .
Hum Gene Ther. 2017 Nov;28(11):964-971. doi: 10.1089/hum.2017.152.
The first attempts at hematopoietic stem cell-based gene therapy (HSC-GT) were reported >25 years ago for primary immune deficiencies, marking the beginning of a vibrant field of translational and therapeutic research. Since then, many HSC-GT studies have been conducted in diverse genetic diseases. The approach has been improved over time, showing biological and therapeutic efficacy with an overall excellent safety record. Within a defined regulatory and ethical landscape, the field of HSC-GT has reached industrialization and commercialization stages, with a landmark recent approval by the European Medicines Agency of the first HSC-GT medicine for human use. At such a pivotal stage, it is important to look back at 25 years of European applied research in this field. This review highlights some of the key contributions of European teams to the field of HSC-GT, focusing in particular on the development of safer gene transfer vectors and international cooperation.
超过 25 年前，人们首次尝试基于造血干细胞的基因治疗（HSC-GT）来治疗原发性免疫缺陷，标志着该领域转化和治疗研究的开始。此后，许多 HSC-GT 研究已在各种遗传疾病中开展。随着时间的推移，该方法不断得到改进，具有良好的生物学和治疗效果，且总体安全性极佳。在明确的监管和伦理框架内，HSC-GT 领域已进入产业化和商业化阶段，最近欧洲药品管理局批准了首款用于人体的 HSC-GT 药物，具有里程碑意义。在这一关键阶段，回顾该领域 25 年来的欧洲应用研究非常重要。本文重点介绍了欧洲团队在 HSC-GT 领域的一些重要贡献，特别是在更安全的基因转移载体的开发和国际合作方面。
