Establishment of the induced pluripotent stem cell line (NCKDi005-A) from a male patient with Alport syndrome carrying a homozygous frameshift mutation in the COL4A4 gene

Stem Cell Res. 2022 Jan:58:102628. doi: 10.1016/j.scr.2021.102628. Epub 2021 Dec 16.

Abstract

Alport syndrome is an inherited chronic kidney disease with genetic heterogeneity. There are three modes of inheritance: X-linked dominant inheritance, autosomal recessive inheritance, and autosomal dominant inheritance. Autosomal recessive inheritance accounts for about 14%-15% of all cases of Alport syndrome and is caused by the COL4A3 or COL4A4 gene mutation. In this study, the peripheral blood mononuclear cells (PBMCs) of a patient with a novel COL4A4 homozygous mutation were reprogrammed into an induced pluripotent stem cell (iPSC) line. The iPSC line can provide a cell model for studying the pathogenesis of the disease and drug screening.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Autoantigens / genetics
  • Collagen Type IV / genetics
  • Frameshift Mutation
  • Humans
  • Induced Pluripotent Stem Cells* / pathology
  • Leukocytes, Mononuclear / pathology
  • Male
  • Mutation / genetics
  • Nephritis, Hereditary* / genetics
  • Pedigree

Substances

  • Autoantigens
  • COL4A4 protein, human
  • Collagen Type IV