Mesenchymal stem cells for trinucleotide repeat disorders.
Mesenchymal stem cells for trinucleotide repeat disorders.
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用于三核苷酸重复疾病的间充质干细胞。
DOI：
10.1007/978-1-62703-411-1_6
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发表时间：
2013
期刊：
影响因子：
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通讯作者：
Nolta,JanA
中科院分区：
文献类型：
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作者：
Annett,Geralyn;Bauer,Gerhard;Nolta,JanA
间充质干细胞/骨髓基质细胞（MSC）非常适合细胞治疗，因为它们易于分离，操作和在临床上的强安全性。它们可以从正常合格的人类供体中大量扩增，并且可以在没有组织匹配的情况下进行输注，因为它们可以保护自己免受免疫系统的影响。无需组织匹配即可移植的能力允许进行大型多中心试验，直接比较相同批次的MSC，而不会出现不良事件或排斥反应。MSC目前已在美国以外的几个国家被批准为药物。MSC可以被遗传修饰以在超生理水平提供生长因子的持续和长期递送。基因修饰的MSC正处于治疗中风的临床试验中，并且正在考虑用于治疗神经退行性疾病如亨廷顿病。
Mesenchymal stem cells/marrow stromal cells (MSCs) are ideally suited for cellular therapy due to their ease of isolation, manipulation, and strong safety profile in the clinic. They can be expanded from normal qualified human donors in large quantities and can be infused without tissue matching, since they shield themselves from the immune system. The ability to be transplanted without tissue matching has allowed large multicenter trials to be conducted with direct comparison of the same batches of MSCs, without adverse events or rejection reactions. MSCs are now approved as drugs in several countries outside of the USA. MSCs can be genetically modified to provide sustained and long-term delivery of growth factors at supraphysiological levels. Gene-modified MSCs are in clinical trials for the treatment of stroke and are under consideration for the treatment of neurodegenerative disorders such as Huntington’s disease.