Article
Partial correction of sensitivity to oxidant stress in Friedreich ataxia patient fibroblasts by frataxin-encoding adeno-associated virus and lentivirus vectors.
Human gene therapy - 1 Aug 2005
Fleming Jane, Spinoulas Afroditi, Zheng Maolin, Cunningham Sharon C, Ginn Samantha L, McQuilty Robert C, Rowe Peter B, Alexander Ian E
Abstract excerpt
Peripheral nervous system (PNS) sensory neurons are directly involved in the pathophysiology of a number of debilitating inherited and acquired neurological conditions. The lack of effective treatments for many such conditions provides a strong rationale for exploring novel therapeutic approaches, including gene therapy. Friedreich ataxia (FRDA), a sensory neuropathy, is a progressive neurodegenerative disease...
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