Generation of four distinct isogenic cell lines with truncating variants in I-band or A-band titin

Truncating variants in TTN (TTNtv) are present in 15–25 % of patients with idiopathic dilated cardiomyopathy. Interestingly, the pathogenicity of TTNtv seems to be linked to their location within the gene. More proximal I-band TTNtv (TTNtvI) harbour less pathogenic potential than distant A-band TTNt...

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Main Authors: Hanne M. Boen, Bert Vandendriessche, Jolien Schippers, Laura Rabaut, Aleksandra Nijak-Paeske, Peter Ponsaerts, Emeline M. Van Craenenbroeck, Bart Loeys, Maaike Alaerts
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Stem Cell Research
Online Access:http://www.sciencedirect.com/science/article/pii/S1873506124002344
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author Hanne M. Boen
Bert Vandendriessche
Jolien Schippers
Laura Rabaut
Aleksandra Nijak-Paeske
Peter Ponsaerts
Emeline M. Van Craenenbroeck
Bart Loeys
Maaike Alaerts
author_facet Hanne M. Boen
Bert Vandendriessche
Jolien Schippers
Laura Rabaut
Aleksandra Nijak-Paeske
Peter Ponsaerts
Emeline M. Van Craenenbroeck
Bart Loeys
Maaike Alaerts
author_sort Hanne M. Boen
collection DOAJ
description Truncating variants in TTN (TTNtv) are present in 15–25 % of patients with idiopathic dilated cardiomyopathy. Interestingly, the pathogenicity of TTNtv seems to be linked to their location within the gene. More proximal I-band TTNtv (TTNtvI) harbour less pathogenic potential than distant A-band TTNtv (TTNtvA). We created isogenic human induced pluripotent stem cell lines (hiPSC) with TTNtvI and TTNtvA using CRISPR/Cas9, for the investigation of the pathomechanism in hiPSC-derived cardiomyocytes (hiPSC-CMs). Exon 48 (E48), located in the I-band, and exon 357 (E357), located in the A-band were targeted.
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publishDate 2024-12-01
publisher Elsevier
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series Stem Cell Research
spelling doaj-art-c3299bc2be0e424f95b05a7926882f592025-08-20T02:38:11ZengElsevierStem Cell Research1873-50612024-12-018110353610.1016/j.scr.2024.103536Generation of four distinct isogenic cell lines with truncating variants in I-band or A-band titinHanne M. Boen0Bert Vandendriessche1Jolien Schippers2Laura Rabaut3Aleksandra Nijak-Paeske4Peter Ponsaerts5Emeline M. Van Craenenbroeck6Bart Loeys7Maaike Alaerts8University of Antwerp and Antwerp University Hospital, GENCOR, Cardiovascular Research, Belgium; University of Antwerp, GENCOR, Center of Medical Genetics, Belgium; Corresponding author.University of Antwerp, GENCOR, Center of Medical Genetics, BelgiumUniversity of Antwerp, GENCOR, Center of Medical Genetics, BelgiumUniversity of Antwerp, GENCOR, Center of Medical Genetics, BelgiumUniversity of Antwerp, GENCOR, Center of Medical Genetics, BelgiumUniversity of Antwerp, Vaccine and Infectious Disease Institute, BelgiumUniversity of Antwerp and Antwerp University Hospital, GENCOR, Cardiovascular Research, BelgiumUniversity of Antwerp, GENCOR, Center of Medical Genetics, BelgiumUniversity of Antwerp, GENCOR, Center of Medical Genetics, BelgiumTruncating variants in TTN (TTNtv) are present in 15–25 % of patients with idiopathic dilated cardiomyopathy. Interestingly, the pathogenicity of TTNtv seems to be linked to their location within the gene. More proximal I-band TTNtv (TTNtvI) harbour less pathogenic potential than distant A-band TTNtv (TTNtvA). We created isogenic human induced pluripotent stem cell lines (hiPSC) with TTNtvI and TTNtvA using CRISPR/Cas9, for the investigation of the pathomechanism in hiPSC-derived cardiomyocytes (hiPSC-CMs). Exon 48 (E48), located in the I-band, and exon 357 (E357), located in the A-band were targeted.http://www.sciencedirect.com/science/article/pii/S1873506124002344
spellingShingle Hanne M. Boen
Bert Vandendriessche
Jolien Schippers
Laura Rabaut
Aleksandra Nijak-Paeske
Peter Ponsaerts
Emeline M. Van Craenenbroeck
Bart Loeys
Maaike Alaerts
Generation of four distinct isogenic cell lines with truncating variants in I-band or A-band titin
Stem Cell Research
title Generation of four distinct isogenic cell lines with truncating variants in I-band or A-band titin
title_full Generation of four distinct isogenic cell lines with truncating variants in I-band or A-band titin
title_fullStr Generation of four distinct isogenic cell lines with truncating variants in I-band or A-band titin
title_full_unstemmed Generation of four distinct isogenic cell lines with truncating variants in I-band or A-band titin
title_short Generation of four distinct isogenic cell lines with truncating variants in I-band or A-band titin
title_sort generation of four distinct isogenic cell lines with truncating variants in i band or a band titin
url http://www.sciencedirect.com/science/article/pii/S1873506124002344
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