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Development of Humanized AAV Vectors for Hemophilia Gene Therapy

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Award date: 
Jun 2, 2015
Award amount: 
Organization name: 
UNC - Chapel Hill
Principal Investigator: 
Li, Chengwen
Grant program category: 
Research and Equipment Support
Grant program name: 
Collaborative Funding Grant
Research Triangle

We will develop AAV vectors for hemophilia gene delivery in mice xenografted with human liver cells. These AAV vectors will have high human liver infection ability and are able to avoid patients' antibody for blocking of AAV infection.

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