Generation of urine-derived iPS cell line via a non-integrative method from a Barth syndrome patient with TAZ gene mutation.

Abstract

Human urine cells from a 6-year-old male X-linked Barth syndrome patient harboring a TAZ frameshift (c.517delG, Xq28) were reprogrammed into the induced pluripotent stem cell (iPSC) line WMUi002-A using non-integration CytoTune®-iPS 2.0 Sendai Virus Reprogramming kit, including four well-known Yamanaka factors SOX2, OCT4, KLF4, and c-MYC. The established patient-derived iPSC expressed endogenous pluripotent markers, had the potential to differentiate into all of the three germ layers, and displayed a normal karyotype.