Abstract：
Genetic manipulation of hematopoietic stem cells (HSCs) is being developed as a therapeutic strategy for several inherited disorders. This field is rapidly evolving with several novel tools and techniques being employed to achieve desired genetic changes. While commercial products are now available for sickle cell disease, transfusion-dependent β-thalassemia, metachromatic leukodystrophy and adrenoleukodystrophy, several challenges remain in patient selection, HSC mobilization and collection, genetic manipulation of stem cells, conditioning, hematologic recovery and post-transplant complications, financial issues, equity of access and institutional and global preparedness. In this report, we explore the current state of development of these therapies and provide a comprehensive assessment of the challenges these therapies face as well as potential solutions.
摘要：
正在开发造血干细胞（HSC）的遗传操作作为几种遗传性疾病的治疗策略。该领域正在迅速发展，采用了几种新颖的工具和技术来实现所需的遗传变化。虽然现在有治疗镰状细胞病的商业产品，输血依赖性β-地中海贫血，异色性脑白质营养不良和肾上腺白质营养不良，在患者选择方面仍然存在一些挑战，HSC动员和收集，干细胞的遗传操作，conditioning,血液学恢复和移植后并发症，财务问题,公平准入以及机构和全球准备。在这份报告中,我们探讨了这些疗法的发展现状,并对这些疗法面临的挑战以及潜在的解决方案进行了全面评估.
