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1 | GLYCODIS3 Genetic correction of glycogen debranching enzyme deficiency in Glycogen Storage disease III: a proof of concept study | 2016 | 173˙076.00 | 173˙076.00 |
2 | DMD2CURE Correction of duplications in the DMD gene by a CRISPR/Cas9 approach | 2017 | 185˙076.00 | 185˙076.00 |
3 | CURECN Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome | 2018 | 6˙249˙103.00 | 6˙249˙103.00 |
4 | MITOPOMPE Targeting mitochondrial defects and oxidative stress in Pompe Disease: from pathogenesis to therapy | 2019 | 173˙076.00 | 173˙076.00 |
The Institution ASSOCIATION GENETHON has been involved also in the following Horizon 2020 projects.