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Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

Adeno-associated viral (AAV) vectors are currently being tested in multiple clinical trials for liver-directed gene transfer to treat the bleeding disorders hemophilia A and B and metabolic disorders. The optimal viral capsid for transduction of human hepatocytes has been under active investigation,...

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Détails bibliographiques
Publié dans:Mol Ther
Auteurs principaux: Vercauteren, Koen, Hoffman, Brad E, Zolotukhin, Irene, Keeler, Geoffrey D, Xiao, Jing W, Basner-Tschakarjan, Etiena, High, Katherine A, Ertl, Hildegund CJ, Rice, Charles M, Srivastava, Arun, de Jong, Ype P, Herzog, Roland W
Format: Artigo
Langue:Inglês
Publié: Nature Publishing Group 2016
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4923326/
https://ncbi.nlm.nih.gov/pubmed/27019999
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.61
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