Efficient Production of Dual Recombinant Adeno-Associated Viral Vectors for Factor VIII Delivery

Recombinant adeno-associated viral (rAAV) vectors have gained attention for human gene therapy because of their high safety and clinical efficacy profile. For factor VIII gene delivery, splitting the coding region between two AAV vectors remains a viable strategy to avoid the packaging capacity limi...

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Autores principales: Wang, Qizhao, Dong, Biao, Firrman, Jenni, Roberts, Sean, Moore, Andrea Rossi, Cao, Wenjing, Diao, Yong, Kapranov, Philipp, Xu, Ruian, Xiao, Weidong
Formato: Artigo
Lenguaje:Inglês
Publicado: Mary Ann Liebert, Inc. 2014
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Research Articles
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC4142791/
https://ncbi.nlm.nih.gov/pubmed/25093498
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hgtb.2014.093
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