CAJ | 학술논문

目的：利用经杆状病毒基因载体系统进行micro-dystrophin基因修饰后的脂肪干细胞(ADSCs)移植治疗Duchenne型肌营养不良症模型(mdx)鼠，探讨ADSCs移植治疗DMD的安全性及可行性。方法 Mdx鼠60只，分为mdx对照组(30只)和mdx移植组(30只)；正常C57小鼠为C57对照组(30只)。体外分离培养小鼠ADSCs，利用杆状病毒基因载体进行micro-dystrophin基因修饰；将基因修饰后的ADSCs经尾静脉移植到mdx鼠体内。于移植后检测mdx鼠的运动功能(采用主动牵引实验和被动转棒实验)、血清CK水平、肌肉病理改变以及肌肉micro-dystrophin表达水平。结果经micro-dystrophin基因修饰的ADSCs移植后，能够重建mdx鼠的micro-dystrophin表达，一定程度上减轻并逆转肌肉的病理损害，进而降低血清CK水平，mdx鼠整体运动功能也有一定改善。结论 ADSCs治疗mdx鼠后，可部分重建模型鼠的dystrophin表达，改善肌肉的病理损害，表明ADSCs是有希望治愈DMD的方法之一。
목적：이용경간상병독기인재체계통진행micro-dystrophin기인수식후적지방간세포(ADSCs)이식치료Duchenne형기영양불량증모형(mdx)서，탐토ADSCs이식치료DMD적안전성급가행성。방법 Mdx서60지，분위mdx대조조(30지)화mdx이식조(30지)；정상C57소서위C57대조조(30지)。체외분리배양소서ADSCs，이용간상병독기인재체진행micro-dystrophin기인수식；장기인수식후적ADSCs경미정맥이식도mdx서체내。우이식후검측mdx서적운동공능(채용주동견인실험화피동전봉실험)、혈청CK수평、기육병리개변이급기육micro-dystrophin표체수평。결과경micro-dystrophin기인수식적ADSCs이식후，능구중건mdx서적micro-dystrophin표체，일정정도상감경병역전기육적병리손해，진이강저혈청CK수평，mdx서정체운동공능야유일정개선。결론 ADSCs치료mdx서후，가부분중건모형서적dystrophin표체，개선기육적병리손해，표명ADSCs시유희망치유DMD적방법지일。
Aim To explore the safety and feasibility of adipose-derived stem cells (ADSCs) transplantation on Duchenne muscular dystrophy (DMD) treatment, in which gene defect on mdx mouse was repaired with recombinant baculovirus carrying micro-dystrophin. Methods Adipose stem cells of mdx mouse were isolated and cultured in vitro. Gene defect was repaired with recombinant baculovirus. The modiifed stem cells were injected DMD mouse model through tail vein. Motor function, serum CK levels, muscle pathology and muscle dystrophin expression were observed after transplantation. Results After transplantation, micro-dystrophin expression in DMD mouse model could be rebuilt, pathological damage on muscles and serum CK levels were reduced, motor function of mouse model showed improvement. Conclusion After transplantation, gene expression can be partially reconstructed, pathological damage can be improved. These results suggested that stem cell transplantation should be a promising cure therapy for DMD.