肠神经嵴干细胞/前体细胞移植重建肠神经系统是治疗肠神经系统疾病的潜在策略，但自体来源的移植细胞增殖、迁徙能力较弱一直是研究的瓶颈。为提高细胞移植效能，本研究拟采用CRISPR/Cas9技术修复RET基因突变HSCR患儿肠管中分离的hENPCs，联合Pepstatin A和（或）5-HT4协同诱导、以及移植前3D水凝胶包裹技术，将hENPCs移植入模型鼠中，评估其移植效果，为实现自体hENPCs移植重建肠神经系统提供理论及实验基础。

The vivo engraftment and migration of enteric neural crest stem cells or enteric neural precursor cells to rescue enteric nervous system is the potential therapy of enteric crest diseases. The capacity of autologous hENCSCs/hENPCs to proliferate and migrate has been limited and may therefore be the choke point to restore the gut function. In this study, we establish a feasible platform to augment the capacity of engrafted cells. CRISPR/Cas9-mediated hENPCs are transplanted into the intestines of the mouse model with the combination of Pepstatin A/5-HT4 confined in a 3D hydrogel to promote the enteric colonization. This study presents cell- and drug-based strategies for the improvement of the capacity of hENPCs and provides the support for the engraftment using autologous source of hENPCs.