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Hemophilia A Gene Therapy: AAV-mediated delivery of an enhanced F8 cassette to the liver produces supraphysiological levels of human FVIII in vivo

 
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Year: 2016

Language: English

Author(s): Brigit E. Riley

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In the Tuesday morning session on gene therapy, Brigit E. Riley  explains her approach to  the AAV-mediated delivery of an enhanced F8 cDNA.