Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8(+) T cells

Recent clinical trials have shown that evasion of CD8(+) T-cell responses against viral capsid is critical for successful liver-directed gene therapy with adeno-associated viral (AAV) vectors for hemophilia. Preclinical models to test whether use of alternate serotypes or capsid variants could avoid...

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Main Authors: Martino, Ashley T., Basner-Tschakarjan, Etiena, Markusic, David M., Finn, Jonathan D., Hinderer, Christian, Zhou, Shangzhen, Ostrov, David A., Srivastava, Arun, Ertl, Hildegund C. J., Terhorst, Cox, High, Katherine A., Mingozzi, Federico, Herzog, Roland W.
格式: Artigo
語言:Inglês
出版: American Society of Hematology 2013
主題:
Gene Therapy
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3606062/
https://ncbi.nlm.nih.gov/pubmed/23325831
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2012-10-460733
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