Abstract
The delta-sarcoglycan (delta-SG) deficient Syrian hamster strain BIO14.6 is one the most used animal model for human primitive cardiomyopathy and muscular dystrophy. The main goal of our project is a rescue of cardiac and muscular functionality of BIO14.6 hamsters using adeno-associated viral (AAV) vectors injection. We observed that therapy could be much more successful in younger animals (16 days old BIO14.6 hamsters), in which we injected 2×1012 GC of the AAV2/8 vector expressing the human delta-SG.
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