Article
Modelling ciliopathy phenotypes in human tissues derived from pluripotent stem cells with genetically ablated cilia.
Nature biomedical engineering - 1 Apr 2022
Cruz Nelly M, Reddy Raghava, McFaline-Figueroa José L, Tran Christine, Fu Hongxia, Freedman Benjamin S
Abstract excerpt
The functions of cilia-antenna-like organelles associated with a spectrum of disease states-are poorly understood, particularly in human cells. Here we show that human pluripotent stem cells (hPSCs) edited via CRISPR to knock out the kinesin-2 subunits KIF3A or KIF3B can be used to model ciliopathy phenotypes and to reveal ciliary functions at the tissue scale. KIF3A-/- and KIF3B-/- hPSCs lacked cilia, yet...
Topics
- Cilia
- Ciliopathies
- Hedgehog Proteins
- Humans
- Kinesins
- Phenotype
- Pluripotent Stem Cells