脂肪组织的干细胞作为神经系统基因治疗载体的研究

目的观察大鼠脂肪组织源性基质细胞表达外源基因的能力和脑内移植后的分布，以获取基因治疗自体移植的载体细胞。方法腺病毒载体Ad5βgal介导法将LacZ转入培养的大鼠脂肪组织源性基质细胞。Hoechst33258标记细胞，立体定向移植到大鼠的纹状体，脑组织切片，在荧光显微镜下检查存活的细胞。结果脂肪组织源性基质细胞能够在体内和体外稳定表达LacZ基因，细胞移植到脑内可以移行，移植细胞没有过渡增生和肿瘤形成，对宿主脑组织无破坏。结论脂肪组织源性基质细胞可以稳定表达夕J源基因，与脑组织有很好的相容性，是中枢神经系统基因治疗的良好载体。

Adipose Tissue-derived Stromal Cells as Vector for Gene Therapy in Central Nervous System

OBJECTIVE: To investigate exogenous gene expressing ability of adipose tissue-derived stromal cell (ADSCs) and cell distribution after they were transplanted into brains, and to get the genetically modified cells for autografting. METHODS: ADSCs were transfected by Ad5beta gal adenovirus containing a report gene, LacZ gene, then they were transplanted into the adult brain of rats, or ADSCs labeled by Hoechst33258 were transplanted into the adult brain of rats to investigate the migration and distribution of cells. RESULTS: ADSCs showed a good expression of LacZ with X-gal staining after transfecting and transplantation into adult brains, and they could incorporate into the host brain tissues and no disruption was observed. These cells showed good compatibility with the host brains. CONCLUSION: The results indicate that ADSCs could incorporate into host brains and express exogenous gene steadily when they were transplanted into adult brain tissues, no overproliferation and gliosis were identified, and ADSCs may be used as a therapeutic gene delivery vehicle in treating CNS disorders in humans.