Effects of FVIII immunity on hepatocyte and hematopoietic stem cell–directed gene therapy of murine hemophilia A

Immune responses to coagulation factors VIII (FVIII) and IX (FIX) represent primary obstacles to hemophilia treatment. Previously, we showed that hematopoietic stem cell (HSC) retroviral gene therapy induces immune nonresponsiveness to FVIII in both naive and preimmunized murine hemophilia A setting...

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Pubblicato in:Mol Ther Methods Clin Dev
Autori principali: Lytle, Allison M, Brown, Harrison C, Paik, Na Yoon, Knight, Kristopher A, Wright, J Fraser, Spencer, H Trent, Doering, Christopher B
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2016
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4750467/
https://ncbi.nlm.nih.gov/pubmed/26909355
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.56
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