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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Detalles Bibliográficos
Autores principales: Okada, Takashi, Takeda, Shin'ichi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2013
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3816704/
https://www.ncbi.nlm.nih.gov/pubmed/24276316
http://dx.doi.org/10.3390/ph6070813

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3816704/
https://www.ncbi.nlm.nih.gov/pubmed/24276316
http://dx.doi.org/10.3390/ph6070813

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