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Characterisation of the patient-specific induced pluripotent stem cell line UKEi075-A carrying the heterozygous PRKAG2 frameshift variant c.769_770del (p.E257Kfs*15)

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Item Type:Article
Title:Characterisation of the patient-specific induced pluripotent stem cell line UKEi075-A carrying the heterozygous PRKAG2 frameshift variant c.769_770del (p.E257Kfs*15)
Creators: Hoffmann, Benedict ORCID logoORCID: https://orcid.org/0009-0008-0165-482X, Knochenhauer, Tim, Schlobohm, Julian ORCID logoORCID: https://orcid.org/0009-0007-9761-3012, Stathopoulou, Konstantina ORCID logoORCID: https://orcid.org/0000-0002-3746-3055, Ulmer, Bärbel M. ORCID logoORCID: https://orcid.org/0000-0001-5712-7270, Shibamiya, Aya, Laufer, Sandra D. ORCID logoORCID: https://orcid.org/0000-0003-3177-7886, Schäfer, Sarina, Krämer, Elisabeth, Fuchs, Sigrid, Augustin, Christa, Girdauskas, Evaldas, Demal, Till J. ORCID logoORCID: https://orcid.org/0000-0001-8978-9914, Klaassen, Sabine ORCID logoORCID: https://orcid.org/0000-0001-5925-9912, Zeller, Tanja, Blankenberg, Stefan ORCID logoORCID: https://orcid.org/0000-0001-6488-2362, Eschenhagen, Thomas ORCID logoORCID: https://orcid.org/0000-0003-1750-1170 and Cuello, Friederike ORCID logoORCID: https://orcid.org/0000-0003-1612-1715
Abstract:Missense variants in PRKAG2, the gene encoding for the γ2-subunit of adenosine monophosphate-activated protein kinase (AMPK), can cause hypertrophic cardiomyopathy, cardiac conduction defects and glycogen storage disorders. Clinical consequences evoked by truncating PRKAG2 variants are poorly understood. Dermal fibroblasts of a male dilated cardiomyopathy-patient carrying the truncating PRKAG2 variant c.769_770del (p.E257Kfs*15) at a heterozygous state were reprogrammed by Sendai virus-mediated expression of OCT-4, SOX2, c-Myc and KLF4 to human induced pluripotent stem cells (hiPSCs). HiPSCs displayed a normal karyotype, high pluripotency, mycoplasma-absence and were successfully differentiated into three primary germ layers. This hiPSC-line will enable analysing molecular pathomechanisms underlying PRKAG2-cardiomyopathy.
Source:Stem Cell Research
ISSN:1873-5061
Publisher:Elsevier
Volume:97
Page Range:104123
Date:December 2026
Official Publication:https://doi.org/10.1016/j.scr.2026.104123
PubMed:View item in PubMed

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