The project aim is to develop brain organoids from Gaucher disease human iPSCs, allowing us to examine the disease progression in vitro.
We plan to develop human brain tissue from Gaucher disease patient-derived stem cells, and then use genetic editing (CRISPR-Cas9) to identify the role of specific genes in this disease. We hope findings from the model allow us to identify new knowledge which will help to develop future therapeutics.
|Gaucher disease organoids
|Effective start/end date
|2021/05/03 → …