Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
作者： Catherine S Manno , Glenn F Pierce , Valder R Arruda , Bertil Glader , Margaret Ragni
DOI: 10.1038/NM1358
关键词: Toxicity 、 Transduction (genetics) 、 Antigen 、 Immunity 、 In vivo 、 Immune system 、 Factor IX 、 Viral vector 、 Medicine 、 Immunology 、 General Biochemistry, Genetics and Molecular Biology 、 General Medicine
摘要: We have previously shown that a single portal vein infusion of a recombinant adeno-associated viral vector (rAAV) expressing canine Factor IX (F.IX) resulted in long-term expression of …
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