Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy
Various characteristics of adeno-associated virus (AAV)-based vectors with long-term safe expression have made it an exciting transduction tool for clinical gene therapy of Duchenne muscular dystrophy (DMD). Although host immune reactions against the vector as well as transgene products were detecte...
| Main Authors: | , |
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| Format: | Online |
| Language: | English |
| Published: |
MDPI
2013
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| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3816704/ |