- An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8
An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8
Molecular therapy. Methods & clinical development (2017-05-10)
Brett Palaschak, Damien Marsic, Roland W Herzog, Sergei Zolotukhin, David M Markusic
PMID28480313
摘要
Multiple independent adeno-associated virus (AAV) gene therapy clinical trials for hemophilia B, utilizing different AAV serotypes, have reported a vector dose-dependent loss of circulating factor IX (FIX) protein associated with capsid-specific CD8
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