Richter Maximilian, Stone Daniel, Miao Carol, Humbert Olivier, Kiem Hans-Peter, Papayannopoulou Thalia, Lieber André
Division of Medical Genetics, University of Washington, 1705 NE Pacific Street, Seattle, WA 98195, USA.
Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue N, Seattle, WA 98109, USA.
Hematol Oncol Clin North Am. 2017 Oct;31(5):771-785. doi: 10.1016/j.hoc.2017.06.001.
Current protocols for hematopoietic stem cell (HSC) gene therapy, involving the transplantation of ex vivo lentivirus vector-transduced HSCs into myeloablated recipients, are complex and not without risk for the patient. In vivo HSC gene therapy can be achieved by the direct modification of HSCs in the bone marrow after intraosseous injection of gene delivery vectors. A recently developed approach involves the mobilization of HSCs from the bone marrow into peripheral the blood circulation, intravenous vector injection, and re-engraftment of genetically modified HSCs in the bone marrow. We provide examples for in vivo HSC gene therapy and discuss advantages and disadvantages.
目前的造血干细胞（HSC）基因治疗方案，包括将体外慢病毒载体转导的造血干细胞移植到接受过骨髓清除的受体中，过程复杂且对患者存在风险。体内造血干细胞基因治疗可通过在骨内注射基因递送载体后直接修饰骨髓中的造血干细胞来实现。最近开发的一种方法涉及将造血干细胞从骨髓动员到外周血液循环中，进行静脉内载体注射，然后将基因修饰的造血干细胞重新植入骨髓。我们提供了体内造血干细胞基因治疗的实例，并讨论了其优缺点。
