Overcoming frataxin gene silencing in Friedreich’s ataxia with small molecules: studies on cellular and animal models
Friedreich’s ataxia (FRDA) is an inherited recessive disorder characterized by progressive neurological disability and heart disease. It is caused by a pathological intronic hyperexpansion of a GAA repeat in the FXN gene, encoding the essential mitochondrial protein frataxin. At the homozygous state...
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| Format: | Others |
| Language: | en |
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Universite Libre de Bruxelles
2010
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| Online Access: | http://theses.ulb.ac.be/ETD-db/collection/available/ULBetd-01152010-173759/ |