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

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...

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Main Authors: Desi Veleva, Merve Ay, Dmitry A. Ovchinnikov, Andrew B.J. Prowse, Minal J. Menezes, Michael Nafisinia
Format: Article
Language:English
Published: Elsevier 2024-06-01
Series:Stem Cell Research
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S1873506124001053
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author Desi Veleva
Merve Ay
Dmitry A. Ovchinnikov
Andrew B.J. Prowse
Minal J. Menezes
Michael Nafisinia
author_facet Desi Veleva
Merve Ay
Dmitry A. Ovchinnikov
Andrew B.J. Prowse
Minal J. Menezes
Michael Nafisinia
author_sort Desi Veleva
collection DOAJ
description 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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spelling doaj.art-eb9b212926cf4db8ad4b0872f9a313382024-03-29T05:49:44ZengElsevierStem Cell Research1873-50612024-06-0177103407Generation of two lymphoblastoid-derived induced pluripotent stem cell (iPSC) lines from patients with phenylketonuriaDesi Veleva0Merve Ay1Dmitry A. Ovchinnikov2Andrew B.J. Prowse3Minal J. Menezes4Michael Nafisinia5StemCore, The Australian Institute for Bioengineering and Nanotechnology, The University of Queensland, St Lucia, QLD, AustraliaStemCore, The Australian Institute for Bioengineering and Nanotechnology, The University of Queensland, St Lucia, QLD, AustraliaThe Florey Melbourne Brain Centre/Kenneth Myer Building, The University of Melbourne, 30 Royal Parade, Melbourne VIC 3010 AustraliaStemCore, The Australian Institute for Bioengineering and Nanotechnology, The University of Queensland, St Lucia, QLD, AustraliaDiscipline of Genetic Medicine, Sydney Medical School, University of Sydney, Sydney, NSW, AustraliaDiscipline 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.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.http://www.sciencedirect.com/science/article/pii/S1873506124001053PhenylketonuriaPKUHuman Induced pluripotent stem cells (hiPSC)High throughput drug screeningPersonalised medicineDrug discovery
spellingShingle Desi Veleva
Merve Ay
Dmitry A. Ovchinnikov
Andrew B.J. Prowse
Minal J. Menezes
Michael Nafisinia
Generation of two lymphoblastoid-derived induced pluripotent stem cell (iPSC) lines from patients with phenylketonuria
Stem Cell Research
Phenylketonuria
PKU
Human Induced pluripotent stem cells (hiPSC)
High throughput drug screening
Personalised medicine
Drug discovery
title Generation of two lymphoblastoid-derived induced pluripotent stem cell (iPSC) lines from patients with phenylketonuria
title_full Generation of two lymphoblastoid-derived induced pluripotent stem cell (iPSC) lines from patients with phenylketonuria
title_fullStr Generation of two lymphoblastoid-derived induced pluripotent stem cell (iPSC) lines from patients with phenylketonuria
title_full_unstemmed Generation of two lymphoblastoid-derived induced pluripotent stem cell (iPSC) lines from patients with phenylketonuria
title_short Generation of two lymphoblastoid-derived induced pluripotent stem cell (iPSC) lines from patients with phenylketonuria
title_sort generation of two lymphoblastoid derived induced pluripotent stem cell ipsc lines from patients with phenylketonuria
topic Phenylketonuria
PKU
Human Induced pluripotent stem cells (hiPSC)
High throughput drug screening
Personalised medicine
Drug discovery
url http://www.sciencedirect.com/science/article/pii/S1873506124001053
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