Modelling CADASIL in a Dish
The Modelling CADASIL in a dish project is run by Dr Alessandra Granata. Dr Granata's research focuses on understanding the mechanisms behind CADASIL, a rare genetic disorder that affects the small blood vessels in the brain, leading to strokes and other neurological issues. To better understand CADASIL, the team take skin cells from patients with CADASIL through a simple biopsy procedure. These skin cells are then reprogrammed into adult stem cells, which have the potential to become any type of cell in the body.
These stem cells are specifically turned into the types of cells found in the small blood vessels of the brain, creating mini-models that mimic the disease's conditions. By studying these models in the lab, Dr Granata and her team can observe how the cells behave and respond to various stimuli, closely simulating what happens in the brains of CADASIL patients. This approach allows identification of potential problems at the cellular level and allows testing of new treatments that could correct these issues. The ultimate goal is to develop effective therapies that can be brought to the clinic, offering hope to those affected by CADASIL and other small vessel diseases.
Dr Granata spoke to us about her work at the CADASIL Meeting 2024, you can watch the talk here.