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首页> 外文期刊>Degenerative Neurological and Neuromuscular Disease >Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy – Phase I–II
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Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy – Phase I–II

机译：动态和非动态性杜氏肌营养不良儿童的干细胞治疗效果– I–II期

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Purpose: Duchenne muscular dystrophy (DMD) is an X-linked recessive pediatric disorder that ultimately leads to progressive muscle degeneration. It has been known that cell-based therapies were used to promote muscle regeneration. The main purpose of this study was to investigate the effects of allogeneic Wharton jelly-derived mesenchymal stem cells therapy in Duchenne muscular dystrophy. Patients and methods: Four ambulatory and five nonambulatory male patients were assessed as having acceptance criteria. Gene expression and immunohistochemical analysis were performed for dystrophin gene expression. The fluorescent in situ hybridization method was used for detection of chimerism and donor–recipient compatibility. Complement dependent lymphocytotoxic crossmatch test and detection of panel reactive antigen were performed. All patients were treated with 2 × 106 cells/kg dose of allogeneic Wharton jelly-derived mesenchymal stem cells via intra-arterial and intramuscular administration. Stability was maintained in patient follow-up tests, which are respiratory capacity tests, cardiac measurements, and muscle strength tests. Results: The vastus intermedius muscle was observed in one patient with MRI. Chimerism was detected by fluorescent in situ hybridization and mean gene expression was increased to 3.3-fold. An increase in muscle strength measurements and pulmonary function tests was detected. Additionally, we observed two of nine patients with positive panel reactive antigen result. Conclusion: All our procedures are well tolerated, and we have not seen any application-related complications so far. Our main purpose of this study was to investigate the effects of allogeneic mesenchymal stem cell therapy and determine its suitability and safety as a form of treatment in this untreatable disorder.
机译：目的：杜兴氏肌营养不良症（DMD）是X连锁隐性儿童疾病，最终导致进行性肌肉变性。已知基于细胞的疗法用于促进肌肉再生。这项研究的主要目的是研究同种异体沃顿胶冻干的间充质干细胞疗法在杜氏肌营养不良症中的作用。患者和方法：将四名卧床和五名非卧床男性患者评估为具有接受标准。进行肌营养不良蛋白基因表达的基因表达和免疫组化分析。荧光原位杂交方法用于检测嵌合体和供体-受体的相容性。进行补体依赖性淋巴细胞毒性交叉匹配测试和面板反应性抗原的检测。所有患者均通过动脉内和肌肉内注射2×106细胞/ kg剂量的同种异体沃顿胶质间充质干细胞进行治疗。在患者随访测试中保持了稳定性，这些随访测试包括呼吸容量测试，心脏测量和肌肉力量测试。结果：1例MRI患者观察到了中间臀大肌。通过荧光原位杂交检测嵌合体，并且平均基因表达增加到3.3倍。检测到肌肉力量测量值和肺功能测试值增加。此外，我们观察到9例面板反应性抗原阳性的患者中有2例。结论：我们所有的程序都被很好地容忍，到目前为止，我们还没有看到任何与应用程序相关的并发症。我们这项研究的主要目的是研究同种异体间充质干细胞疗法的效果，并确定其作为这种不可治愈疾病的一种治疗形式的适用性和安全性。

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