The lab of nuclease enabled cell therapies at VU EMBL partnership institute for gene editing technologies, Jonathan Arias (PhD) Principal Investigator.

The convergence between human iPS cells and gene editing technologies allow us to access cell types and genome configurations that are rare or normally inaccessible for diseasemodelling and therapeutic prototyping ex vivo.

During this talk I will present developments for the creation of isogenic cell models using bi-allelic editing with CRISPR-Cas9 in a deterministic manner. I will discuss how such bi-allelic editing can be used to model neurodegeneration in Parkinson’s disease and the early onset rare-disease ceroid lipofuscinosis.

Furthermore, I will show how genetically encoded sensors enable the stratification of patients and facilitates compound screening with high-throughput and high-content systems.