Generation of two lymphoblastoid-derived induced pluripotent stem cell (iPSC) lines from patients with phenylketonuria.

Stem Cell Res

Discipline of Genetic Medicine, Sydney Medical School, University of Sydney, Sydney, NSW, Australia; Storr Liver Centre, Westmead Institute for Medical Research,Sydney, NSW, Australia. Electronic address:

Published: June 2024

We employed a Sendai virus-based reprogramming method to transform human lymphoblastoid cell lines (LCL) derived from two individuals diagnosed with phenylketonuria (PKU) into induced pluripotent stem cells (iPSC). This reprogramming process involved the expression of the four Yamanaka factors: KLF4, OCT4, SOX2, and C-MYC. The resulting patient-specific iPSCs exhibited a normal karyotype and expressed endogenous pluripotent markers NANOG and OCT-4. Notably, these iPSCs demonstrated strong differentiation capabilities, giving rise to cell populations representing the ectoderm, endoderm, and mesoderm germ layers.

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http://dx.doi.org/10.1016/j.scr.2024.103407DOI Listing

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