CORDIS Project
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This project aims to develop gene therapy for Glycogen Storage Disease III, a rare genetic disorder. Using a mouse model, it explores two therapeutic strategies to restore the function of the glycogen debranching enzyme, focusing on both liver and muscle tissues.
Glycogen storage disease III (GSD III) is a rare (1:100,000) autosomal recessive disorder that results from the deficiency of the glycogen debranching enzyme (GDE).
The major cause of morbidity is associated with the muscle accumulation of glycogen, which leads to progressive myopathy. A dietary treatment with frequent meals high in carbohydrates, slows the progression of the pathology that is however inevitable.
There is no cure for GSD III, the recent development of a mouse model lacking GDE a…
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