Center for Cell Engineering, Molecular Pharmacology and Chemistry Program, Memorial Sloan-Kettering Cancer Center, New York, NY, USA.
Blood. 2012 Feb 2;119(5):1107-16. doi: 10.1182/blood-2011-09-349993. Epub 2011 Nov 17.
The genetic engineering of hematopoietic stem cells is the basis for potentially treating a large array of hereditary and acquired diseases, and stands as the paradigm for stem cell engineering in general. Recent clinical reports support the formidable promise of this approach but also highlight the limitations of the technologies used to date, which have on occasion resulted in clonal expansion, myelodysplasia, or leukemogenesis. New research directions, predicated on improved vector designs, targeted gene delivery or the therapeutic use of pluripotent stem cells, herald the advent of safer and more effective hematopoietic stem cell therapies that may transform medical practice. In this review, we place these recent advances in perspective, emphasizing the solutions emerging from a wave of new technologies and highlighting the challenges that lie ahead.
造血干细胞的基因工程是治疗多种遗传性和获得性疾病的基础，也是干细胞工程的典范。最近的临床报告支持了这种方法的巨大潜力，但也强调了迄今为止所使用技术的局限性，这些技术有时会导致克隆扩增、骨髓增生异常或白血病发生。基于改进的载体设计、靶向基因传递或多能干细胞的治疗应用的新研究方向预示着更安全、更有效的造血干细胞治疗方法的出现，这可能会改变医学实践。在这篇综述中，我们从新的视角看待这些最新进展，强调新技术浪潮带来的解决方案，并突出未来的挑战。
