High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines

Elimination of specific surface-exposed single tyrosine (Y) residues substantially improves hepatic gene transfer with adeno-associated virus type 2 (AAV2) vectors. Here, combinations of mutations in the seven potentially relevant Y residues were evaluated for further augmentation of transduction ef...

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Bibliographische Detailangaben
Hauptverfasser: Markusic, David M, Herzog, Roland W, Aslanidi, George V, Hoffman, Brad E, Li, Baozheng, Li, Mengxin, Jayandharan, Giridhara R., Ling, Chen, Zolotukhin, Irene, Ma, Wenqin, Zolotukhin, Sergei, Srivastava, Arun, Zhong, Li
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2010
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2997584/
https://ncbi.nlm.nih.gov/pubmed/20736929
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.172
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