GENEFA is born with the sole purpose of gathering all efforts to achieve a cure for FA.
This way, people attached to our platform opted for the following project:
This project has been evaluated as the best stand of all, since it takes into account the corrected gene which has shown to be functionally transcribed and long-lasting; therefore it only remains studying the insertion procedure for the corrected gene to cross the blood-brain barrier and improving its diffussion.
The minimum budget for carrying out the project is approximately € 100,000 per year over three years. Being a rare disease with few affected people, the economic burden on their families is very large and that is why we launched this appeal, because gathering all efforts we would be able to carry out this research and furthermore contribute to scientific development to cure genetic diseases as a whole.