Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids

Vectors based on the clade E family member adeno-associated virus (AAV) serotype 8 have shown promise in patients with hemophilia B and have emerged as best in class for human liver gene therapies. We conducted a thorough evaluation of liver-directed gene therapy using vectors based on several natur...

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Detalles Bibliográficos
Publicado en:Mol Ther
Main Authors: Wang, Lili, Bell, Peter, Somanathan, Suryanarayan, Wang, Qiang, He, Zhenning, Yu, Hongwei, McMenamin, Deirdre, Goode, Tamara, Calcedo, Roberto, Wilson, James M
Formato: Artigo
Idioma:Inglês
Publicado: Nature Publishing Group 2015
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Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4700115/
https://ncbi.nlm.nih.gov/pubmed/26412589
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.179
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