Crowdfunding the Development of a therapy for Friedreich Ataxia
Friedreich Ataxia is due to an expansion in the number of triplets of nucleotides (GAA) in the gene sequence which codes for the production of frataxin protein. In a healthy person there are 8-34 GAA repeats (GAA-GAA-GAA-GAA-GAA ……). However, for most patients, there are several hundreds GAA repeats. This mutation...