Reshaping AAV vectors for liver gene therapy
نویسندگان
چکیده
منابع مشابه
Pre-clinical in vivo evaluation of pseudotyped adeno-associated virus (AAV) vectors for liver gene therapy
We report the generation and use of pseudotyped adeno-associated viral (AAV) vectors for the liver-specific expression of human blood coagulation Factor IX (hFIX). Therefore, an AAV-2 genome encoding the hfIX gene was cross-packaged into capsids of AAV types 1 to 6, using efficient large-scale technology for particle production and purification. In immunocompetent mice, the resulting vector par...
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ژورنال
عنوان ژورنال: Hepatology
سال: 2008
ISSN: 0270-9139
DOI: 10.1002/hep.22590