Undetectable Transcription of cap in a Clinical AAV Vector: Implications for Preformed Capsid in Immune Responses

In a gene therapy clinical trial for hemophilia B, adeno-associated virus 2 (AAV2) capsid–specific CD8(+) T cells were previously implicated in the elimination of vector-transduced hepatocytes, resulting in loss of human factor IX (hFIX) transgene expression. To test the hypothesis that expression o...

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Main Authors: Hauck, Bernd, Murphy, Samuel L, Smith, Peter H, Qu, Guang, Liu, Xingge, Zelenaia, Olga, Mingozzi, Federico, Sommer, Jürg M, High, Katherine A, Wright, J. Fraser
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2008
Teme:
Original Articles
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC2834997/
https://ncbi.nlm.nih.gov/pubmed/18941440
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.227
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