Generation of induced pluripotent stem cells (NJDTHi001-A) from a Danon disease child with mutation of c.467 T > G in LAMP2 gene.

Stem Cell Res

Department of Cardio-Thoracic Surgery, Nanjing Drum Tower Hospital, The Affiliated Hospital of Nanjing University Medical School, Nanjing, Jiangsu Province, China. Electronic address:

Published: December 2020

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.

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http://dx.doi.org/10.1016/j.scr.2020.101994DOI Listing

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