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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Bibliografiske detaljer
Main Authors: Wang, Qizhao, Dong, Biao, Firrman, Jenni, Roberts, Sean, Moore, Andrea Rossi, Cao, Wenjing, Diao, Yong, Kapranov, Philipp, Xu, Ruian, Xiao, Weidong
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2014
Fag:
Research Articles
Online adgang: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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