In vitro retinal disease modelling for retinal therapy
Newcells offers a fast and reliable in vitro safety and efficacy service for the evaluation of novel compounds for retinal therapy using complex human retinal organoid or RPE models developed in house. Both models are iPSC derived allowing gene-edited lines to be engineered using CRISPR/Cas9 for direct comparison between WT and mutant phenotypes after differentiation. Targeted mutations can model retinopathies, more specifically monogenic inherited retinopathies such as retinitis pigmentosa (RP), Stargardt disease, Usher’s syndrome and Leber congenital amaurosis.
Service outputs
- Phenotypic comparison of WT and gene-edited retinal organoids or RPE cells
- Analysis of key markers by qualitative immunofluorescence
- Assessment of cell type composition of the retinal organoids using imaging techniques
- Evaluation of photoreceptor degeneration
- Loss of RPE barrier function analysis
- Loss of phagocytosis of photoreceptor outer segment in RPE analysis
- Time points comparisons (D60, D120, D150, D210)