Superior human hepatocyte transduction with Adeno-associated Virus Vector Serotype 7

Although therapeutic outcomes have been achieved in hemophilia patients after delivery of clotting factor genes to the liver using adeno-associated virus (AAV) vectors, it is well known that the pre-clinical results generated from hemophilia animal models have not been directly predictive of success...

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Veröffentlicht in:Gene Ther
Hauptverfasser: Shao, Wenwei, Pei, Xiaolei, Cui, Caibin, Askew, Charles, Dobbins, Amanda, Chen, Xiaojing, Abajas, Yasmina L., Gerber, David A., Samulski, R. Jude, Nichols, Timothy C., Li, Chengwen
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2019
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6923567/
https://ncbi.nlm.nih.gov/pubmed/31570819
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41434-019-0104-5
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