Generation of an induced pluripotent stem cell line (SDASi001-A) from a Schimke immune-osseous dysplasia patient with SMARCAL1 mutations

Stem Cell Res. 2023 Oct:72:103217. doi: 10.1016/j.scr.2023.103217. Epub 2023 Sep 25.

Abstract

A human induced pluripotent stem cell (iPSC) line (SDASi001-A) was generated from patient with Schimke immune-osseous dysplasia (SIOD), carrying heterozygous mutations in SMARCAL1 gene. Peripheral blood mononuclear cells (PBMCs) were reprogrammed using non-integrating delivery of OCT4, SOX2, KFL4, BCL-XL and c-MYC. The iPSC line expresses pluripotency markers, displays a normal karyotype, and has the ability to differentiate into cells of three germ layers in vitro. This iPSC line represents a valuable cell model for SIOD in humans.

MeSH terms

  • Cell Differentiation
  • DNA Helicases / genetics
  • DNA Helicases / metabolism
  • Heterozygote
  • Humans
  • Induced Pluripotent Stem Cells* / metabolism
  • Kruppel-Like Factor 4
  • Leukocytes, Mononuclear / metabolism
  • Mutation / genetics

Substances

  • Kruppel-Like Factor 4
  • SMARCAL1 protein, human
  • DNA Helicases