Increasing the expression of frataxin with the CRISPR system
Friedreich is an hereditary disease, which in due to a mutation of the frataxin gene. This mutation leads to a reduced production of the frataxin protein. This leads to oxidative stress and death of the cells. The death of the neurons and cardiomyocytes are responsible for progressive severe neurological and cardiac symptoms. The aim of the research project is to increase the expression of frataxin by targeting the promoter with a gRNA that will form a complex with a Cas9-VP64 protein leading to an increase expression of the frataxin gene that will hopefully reduce the symptoms of the disease. Experiments will be conducted in cells of Friedreich patients and in a mouse model of the disease.