CORDIS Project
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This project focuses on developing therapies for Friedreich’s Ataxia, a genetic disorder that affects movement coordination and heart function. It aims to increase levels of the frataxin protein, which is crucial for mitochondrial health, by preventing its degradation through small molecules that inhibit ubiquitination…
Friedreich’s Ataxia (FRDA) is a devastating degenerative disease with no specific therapy.
It is passed by autosomal recessive inheritance and affects 1:30,000 individuals in Caucasian populations.
Symptoms appear in the first decade of life and include progressive and unremitting lack of movement coordination, leading to complete inability, and dilated cardiomyopathy leading to congestive heart failure, the most common cause of premature death. FRDA is due to the insufficient transcription of t…
UNIVERSITA DEGLI STUDI DI ROMA TOR VERGATA
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