Generation of the CRISPR/Cas9-mediated KIF1C knock-out human iPSC line HIHRSi003-A-1

Stem Cell Res. 2020 Dec:49:102059. doi: 10.1016/j.scr.2020.102059. Epub 2020 Oct 29.

Abstract

Bi-allelic loss-of-function mutations in the gene encoding the motor protein KIF1C are associated with Hereditary Spastic Paraplegia (HSP) type SPG58, a slowly progressive neurodegenerative motoneuron disease. The biological role of KIF1C is incompletely understood. We used a protein-based CRISPR/Cas9 genome editing approach to generate a homozygous KIF1C knock-out iPSC line (HIHRSi003-A-1) from a healthy control. This iPSC-KIF1C-/- line and the corresponding isogenic control are a useful model to study the physiological function of KIF1C and the pathophysiological consequences of KIF1C dysfunction in human disease.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • CRISPR-Cas Systems / genetics
  • Gene Editing
  • Homozygote
  • Humans
  • Induced Pluripotent Stem Cells*
  • Kinesins / genetics
  • Spastic Paraplegia, Hereditary* / genetics

Substances

  • KIF1C protein, human
  • Kinesins