Friedreich Ataxia: Treatment with Genetic Approach

Abstract

Friedreich ataxia (FA) is a disorder in the nervous system inherited according to the Mendel’s first law. Mutations in the FXN gene trigger the FA disorder. The FXN gene occupies chromosome 9q21.11 in the chromosome map. Mutations in the FXN gene consist of four classes of alleles. These include normal alleles, changeable normal alleles, complete penetrance alleles, and borderline alleles. Currently, there is no efficient treatment for this disorder. To slow down FA, genetic approach can be used. The approach may comprise genetic counseling and use of gene therapy. In genetic counseling, if both parents are carriers, a child has a 25 % FA. To detect people with carrier, amniocentesis can be used for instance. Preimplantation FA diagnostic can also be made. In gene therapy, DNA banking is needed. It is useful to study Friedreich ataxia for treatment of this disorder. Gene therapy is a method to correct damaged cells of patients. In this article, the author shows viral vectors such as HSV-1 amplicon vectors and lentivirus vectors as gene shipping vehicles. Both can restore the normal point of frataxin. These results show that gene therapy is a promising tool to treat FA disorder.