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Methods of generating stem cells and embryonic bodies carrying disease-causing mutations and methods of using same for studying genetic disorders

机译:带有致病突变的干细胞和胚胎体的产生方法及其用于研究遗传疾病的方法

摘要

Stem cells, stem cell lines and differentiated cells, tissues and organs which carry disease-causing mutations are provided. There is also provided a method of identifying agents suitable for treating disorders associated with at least one disease-causing mutations such as myotonic dystrophy and van Waardenburg syndrome.
机译:提供了带有致病突变的干细胞,干细胞系以及分化的细胞,组织和器官。还提供了一种鉴定适于治疗与至少一种引起疾病的突变有关的疾病的试剂的方法,所述疾病例如肌强直性营养不良和范瓦登堡综合症。

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