Periodic Reporting for period 1 - GLYCODIS3 (Genetic correction of glycogen debranching enzyme deficiency in Glycogen Storage disease III: a proof of concept study)

Glycogen storage disease III (GSDIII) is a rare (1:100,000) autosomal recessive disorder resulting from the deficiency of glycogen debranching enzyme (GDE) the enzyme that hydrolyzes glycogen branches in the cytosol. The disease manifestation occurs in two phases. Early...