Transcriptional profiles of microglia-like progeny cells derived from Cx3cr1 haplo-insufficient HSPCs and wild-tyoe HSPCs

Favoring the engraftment and maturation of the engineered HSPCs in the central nervous system could allow enhancing further the therapeutic potential of the transplantation of engineered HSPCs for treating neurometabolic diseases. We propose a gene addition strategy involving Cx3cr1, a microglia chemokine receptor regulating microglia ontogeny and function. Here we characterize the transcriptional profile of microglia-like cell generated by Cx3cr1 haplo-insufficient and WT HSPC Overall design: Microglia-like cell generated by Cx3cr1 haplo-insufficient and WT HSPC were isolated from the CNS of conditioned mice

若能促进工程化造血干细胞与祖细胞（hematopoietic stem and progenitor cells, HSPCs）在中枢神经系统（central nervous system, CNS）内的植入与成熟，便可进一步提升工程化HSPC移植治疗神经代谢疾病的治疗潜力。本研究提出一项基于Cx3cr1的基因添加策略：Cx3cr1是调控小胶质细胞发育与功能的小胶质细胞趋化因子受体。本研究对由Cx3cr1单倍剂量不足型与野生型（wild type, WT）HSPC诱导生成的类小胶质细胞的转录谱进行了表征。实验设计概述：从经预处理小鼠的中枢神经系统中，分离得到由Cx3cr1单倍剂量不足型与野生型HSPC诱导生成的类小胶质细胞。