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Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models

Vectors based on adeno-associated viruses (AAVs) are being evaluated for use in liver-directed gene therapy. Candidates have been preselected on the basis of capsid structure that plays an important role in determining performance profiles. We describe a comprehensive and statistically powered set o...

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Main Authors: Wang, Lili, Wang, Huan, Bell, Peter, McCarter, Robert J, He, Jianping, Calcedo, Roberto, Vandenberghe, Luk H, Morizono, Hiroki, Batshaw, Mark L, Wilson, James M
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2009
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC2839210/
https://ncbi.nlm.nih.gov/pubmed/19861950
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.246
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