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High density SNP array and reanalysis of genome sequencing uncovers CNVs associated with neurodevelopmental disorders in KOLF2.1J iPSCs

The KOLF2.1J iPSC line was recently proposed as a reference iPSC to promote the standardization of research studies in the stem cell field. Due to overall good performance differentiating to neural cell lineages, high gene editing efficiency, and absence of genetic variants associated to neurologica...

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Autores principales: Gracia-Diaz, Carolina, Perdomo, Jonathan E., Khan, Munir E., Disanza, Brianna, Cajka, Gregory G., Lei, Sunyimeng, Gagne, Alyssa, Maguire, Jean Ann, Roule, Thomas, Shalem, Ophir, Bhoj, Elizabeth J., Ahrens-Nicklas, Rebecca C., French, Deborah, Goldberg, Ethan M., Wang, Kai, Glessner, Joseph, Akizu, Naiara
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10327134/
https://www.ncbi.nlm.nih.gov/pubmed/37425875
http://dx.doi.org/10.1101/2023.06.26.546614
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author Gracia-Diaz, Carolina
Perdomo, Jonathan E.
Khan, Munir E.
Disanza, Brianna
Cajka, Gregory G.
Lei, Sunyimeng
Gagne, Alyssa
Maguire, Jean Ann
Roule, Thomas
Shalem, Ophir
Bhoj, Elizabeth J.
Ahrens-Nicklas, Rebecca C.
French, Deborah
Goldberg, Ethan M.
Wang, Kai
Glessner, Joseph
Akizu, Naiara
author_facet Gracia-Diaz, Carolina
Perdomo, Jonathan E.
Khan, Munir E.
Disanza, Brianna
Cajka, Gregory G.
Lei, Sunyimeng
Gagne, Alyssa
Maguire, Jean Ann
Roule, Thomas
Shalem, Ophir
Bhoj, Elizabeth J.
Ahrens-Nicklas, Rebecca C.
French, Deborah
Goldberg, Ethan M.
Wang, Kai
Glessner, Joseph
Akizu, Naiara
author_sort Gracia-Diaz, Carolina
collection PubMed
description The KOLF2.1J iPSC line was recently proposed as a reference iPSC to promote the standardization of research studies in the stem cell field. Due to overall good performance differentiating to neural cell lineages, high gene editing efficiency, and absence of genetic variants associated to neurological disorders KOLF2.1J iPSC line was particularly recommended for neurodegenerative disease modeling. However, our work uncovers that KOLF2.1J hPSCs carry heterozygous small copy number variants (CNVs) that cause DTNBP1, JARID2 and ASTN2 haploinsufficiencies, all of which are associated with neurological disorders. We further determine that these CNVs arose in vitro over the course of KOLF2.1J iPSC generation from a healthy donor-derived KOLF2 iPSC line and affect the expression of DNTBP1, JARID2 and ASTN2 proteins in KOLF2.1J iPSCs and neural progenitors. Therefore, our study suggests that KOLF2.1J iPSCs carry genetic variants that may be deleterious for neural cell lineages. This data is essential for a careful interpretation of neural cell studies derived from KOLF2.1J iPSCs and highlights the need for a catalogue of iPSC lines that includes a comprehensive genome characterization analysis.
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spelling pubmed-103271342023-07-08 High density SNP array and reanalysis of genome sequencing uncovers CNVs associated with neurodevelopmental disorders in KOLF2.1J iPSCs Gracia-Diaz, Carolina Perdomo, Jonathan E. Khan, Munir E. Disanza, Brianna Cajka, Gregory G. Lei, Sunyimeng Gagne, Alyssa Maguire, Jean Ann Roule, Thomas Shalem, Ophir Bhoj, Elizabeth J. Ahrens-Nicklas, Rebecca C. French, Deborah Goldberg, Ethan M. Wang, Kai Glessner, Joseph Akizu, Naiara bioRxiv Article The KOLF2.1J iPSC line was recently proposed as a reference iPSC to promote the standardization of research studies in the stem cell field. Due to overall good performance differentiating to neural cell lineages, high gene editing efficiency, and absence of genetic variants associated to neurological disorders KOLF2.1J iPSC line was particularly recommended for neurodegenerative disease modeling. However, our work uncovers that KOLF2.1J hPSCs carry heterozygous small copy number variants (CNVs) that cause DTNBP1, JARID2 and ASTN2 haploinsufficiencies, all of which are associated with neurological disorders. We further determine that these CNVs arose in vitro over the course of KOLF2.1J iPSC generation from a healthy donor-derived KOLF2 iPSC line and affect the expression of DNTBP1, JARID2 and ASTN2 proteins in KOLF2.1J iPSCs and neural progenitors. Therefore, our study suggests that KOLF2.1J iPSCs carry genetic variants that may be deleterious for neural cell lineages. This data is essential for a careful interpretation of neural cell studies derived from KOLF2.1J iPSCs and highlights the need for a catalogue of iPSC lines that includes a comprehensive genome characterization analysis. Cold Spring Harbor Laboratory 2023-06-26 /pmc/articles/PMC10327134/ /pubmed/37425875 http://dx.doi.org/10.1101/2023.06.26.546614 Text en https://creativecommons.org/licenses/by-nc-nd/4.0/This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (https://creativecommons.org/licenses/by-nc-nd/4.0/) , which allows reusers to copy and distribute the material in any medium or format in unadapted form only, for noncommercial purposes only, and only so long as attribution is given to the creator.
spellingShingle Article
Gracia-Diaz, Carolina
Perdomo, Jonathan E.
Khan, Munir E.
Disanza, Brianna
Cajka, Gregory G.
Lei, Sunyimeng
Gagne, Alyssa
Maguire, Jean Ann
Roule, Thomas
Shalem, Ophir
Bhoj, Elizabeth J.
Ahrens-Nicklas, Rebecca C.
French, Deborah
Goldberg, Ethan M.
Wang, Kai
Glessner, Joseph
Akizu, Naiara
High density SNP array and reanalysis of genome sequencing uncovers CNVs associated with neurodevelopmental disorders in KOLF2.1J iPSCs
title High density SNP array and reanalysis of genome sequencing uncovers CNVs associated with neurodevelopmental disorders in KOLF2.1J iPSCs
title_full High density SNP array and reanalysis of genome sequencing uncovers CNVs associated with neurodevelopmental disorders in KOLF2.1J iPSCs
title_fullStr High density SNP array and reanalysis of genome sequencing uncovers CNVs associated with neurodevelopmental disorders in KOLF2.1J iPSCs
title_full_unstemmed High density SNP array and reanalysis of genome sequencing uncovers CNVs associated with neurodevelopmental disorders in KOLF2.1J iPSCs
title_short High density SNP array and reanalysis of genome sequencing uncovers CNVs associated with neurodevelopmental disorders in KOLF2.1J iPSCs
title_sort high density snp array and reanalysis of genome sequencing uncovers cnvs associated with neurodevelopmental disorders in kolf2.1j ipscs
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10327134/
https://www.ncbi.nlm.nih.gov/pubmed/37425875
http://dx.doi.org/10.1101/2023.06.26.546614
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