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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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Main Authors: Shin'ichi Takeda, Takashi Okada
Formato: Artigo
Idioma:Inglês
Publicado em: MDPI AG 2013-06-01
Colecção:Pharmaceuticals
Assuntos:
DMD
AAV
Acesso em linha:http://www.mdpi.com/1424-8247/6/7/813
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