Article
Functional recovery in a Friedreich's ataxia mouse model by frataxin gene transfer using an HSV-1 amplicon vector.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jun 2007
Lim Filip, Palomo Gloria M, Mauritz Christina, Giménez-Cassina Alfredo, Illana Belen, Wandosell Francisco, Díaz-Nido Javier
Abstract excerpt
There is currently no effective treatment for Friedreich's ataxia (FA), the most common of the hereditary ataxias. The disease is caused by mutations in FRDA that drastically reduce expression levels of the mitochondrial protein frataxin. In FA animal models, a key difficulty is obtaining the precise levels of frataxin expression in the appropriate tissues to provoke pathology without early lethality. To develop...
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