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Projects. Life and Matter Sciences

Phosphodiesterase inhibitors as Friedreich's ataxia treatment

Lead Researcher: Pilar González Cabo
Research Centre: Centro de Investigación Biomédica en Red (CIBER). Departamento de Fisiología, Facultad de Medicina y Odontología. Universitat de València.


Pilar González CaboFriedreich's ataxia (FRDA) is a peripheral neuropathy with autosomal recessive inheritance caused by a deficit in the levels of a mitochondrial protein called frataxin (FXN). This rare neurodegenerative disease is characterized by progressive gait ataxia associated with hypertrophic cardiomyopathy and diabetes mellitus. To date, there is no FRDA treatment able to alter the natural history of this neurological disease.

Previously, we have studied the effectiveness of phosphodiesterase (PDE) inhibitors as a novel pharmacological approach against axonal degeneration of sensory neurons in an FRDA mouse model. The treatment, in a primary culture of sensory neurons, improves the morphology of the mitochondrial network and reverts axonal degeneration. In addition, in the FRDA Drosophila model (fruit fly), this treatment rescues the diminished motor capacity, reaching normal levels. Therefore, our proposal is to carry out a pre-clinical trial with PDE inhibitors in a mouse model of the disease. The project also contemplates the identification of the biochemical and molecular mechanism of action of PDE inhibitors. With this aim, a screening of candidate genes will be carried out in the Drosophila model.

The results of this project will have a clear clinical benefit: the possibility of proposing phosphodiesterase inhibitors as a pharmacological strategy for clinical trials in FRDA.

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