The induced pluripotent stem cells (iPSCs) were generated from peripheral blood mononuclear cells (PBMCs) isolated from a male child with Danon disease. Next-generation sequencing (NGS) revealed a stop gained mutation c.467 T > G in LAMP2 gene. PBMCs were reprogrammed by using non-integrating Sendai reprogramming kits. It is a possible cell model to screen alternative medicine treatment of the Danon disease.

中文翻译：

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从达农氏病患儿中产生诱导多能干细胞（NJDTHi001-A），LAMP2基因中的c.467 T> G突变

诱导的多能干细胞（iPSC）由分离自患有Danon病的男性儿童的外周血单核细胞（PBMC）产生。下一代测序（NGS）显示了LAMP2基因中的一个停止获得的突变c.467 T>G。通过使用非集成的仙台重编程工具包对PBMC进行重编程。筛选达农病的替代药物治疗可能是细胞模型。