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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...

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Autors principals: Okada, Takashi, Takeda, Shin'ichi
Format: Artigo
Idioma:Inglês
Publicat: MDPI 2013
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3816704/
https://ncbi.nlm.nih.gov/pubmed/24276316
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ph6070813
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