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Generation of induced pluripotent stem cell (iPSC) lines carrying a heterozygous (UKWMPi002-A-1) and null mutant knockout (UKWMPi002-A-2) of Cadherin 13 associated with neurodevelopmental disorders using CRISPR/Cas9

  • Maria Rosaria Vitale*
  • , Johanna Eva Maria Zoeller
  • , Charline Jansch
  • , Anna Janz
  • , Frank Edenhofer
  • , Eva Klopocki
  • , Daniel van den Hove
  • , Tim Vanmierlo
  • , Olga Rivero
  • , Nael Nadif Kasri
  • , Georg Christoph Ziegler
  • , Klaus-Peter Lesch*
  • *Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

Abstract

Fibroblasts isolated from a skin biopsy of a healthy 46-year-old female were infected with Sendai virus containing the Yamanaka factors to produce transgene-free human induced pluripotent stem cells (iPSCs). CRISPR/Cas9 was used to generate isogenic cell lines with a gene dose-dependent deficiency of CDH13, a risk gene associated with neurodevelopmental and psychiatric disorders. Thereby, a heterozygous CDH13 knockout (CDH13(+/-)) and a CDH13 null mutant (CDH13(-/-)) iPSC line was obtained. All three lines showed expression of pluripotency-associated markers, the ability to differentiate into cells of the three germ layers in vitro, and a normal female karyotype.

Original languageEnglish
Article number102169
Pages (from-to)1-5
Number of pages5
JournalStem Cell Research
Volume51
DOIs
Publication statusPublished - Mar 2021

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