Epigenetic modification of the frataxin gene for the treatment of Friedreich’s ataxia
<p>Friedreich's Ataxia (FRDA) is a recessive neurodegenerative disease caused by the partial silencing of the frataxin gene (FXN). Long GAA triplet repeat expansions in the first intron of FXN are responsible for the reduced levels of frataxin, an essential mitochondrial protein involved...
| Main Author: | |
|---|---|
| Other Authors: | |
| Format: | Thesis |
| Published: |
2018
|
| Subjects: |