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

Desi Veleva; Merve Ay; Dmitry A. Ovchinnikov; Andrew B.J. Prowse; Minal J. Menezes; Michael Nafisinia

Stem Cell Research (Jun 2024)

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

Desi Veleva,
Merve Ay,
Dmitry A. Ovchinnikov,
Andrew B.J. Prowse,
Minal J. Menezes,
Michael Nafisinia

Affiliations

Desi Veleva: StemCore, The Australian Institute for Bioengineering and Nanotechnology, The University of Queensland, St Lucia, QLD, Australia
Merve Ay: StemCore, The Australian Institute for Bioengineering and Nanotechnology, The University of Queensland, St Lucia, QLD, Australia
Dmitry A. Ovchinnikov: The Florey Melbourne Brain Centre/Kenneth Myer Building, The University of Melbourne, 30 Royal Parade, Melbourne VIC 3010 Australia
Andrew B.J. Prowse: StemCore, The Australian Institute for Bioengineering and Nanotechnology, The University of Queensland, St Lucia, QLD, Australia
Minal J. Menezes: Discipline of Genetic Medicine, Sydney Medical School, University of Sydney, Sydney, NSW, Australia
Michael Nafisinia: Discipline of Genetic Medicine, Sydney Medical School, University of Sydney, Sydney, NSW, Australia; Storr Liver Centre, Westmead Institute for Medical Research,Sydney, NSW, Australia; Corresponding author at: Storr Liver Centre, Westmead Institute for Medical Research, 176 Hawkesbury Rd, Westmead, NSW 2145, Australia.

Journal volume & issue: Vol. 77
p. 103407

Abstract

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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.

Published in Stem Cell Research

ISSN: 1873-5061 (Print); 1876-7753 (Online)
Publisher: Elsevier
Country of publisher: Netherlands
LCC subjects: Science: Biology (General)
Website: https://www.journals.elsevier.com/stem-cell-research

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