Liver-Directed Gene Therapy for Hemophilia with Immune Shielded Lentiviral Vectors


Year: 2018

Language: English

Author(s): Alessio Cantore

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In the Wednesday afternoon session entitled Gene Therapy – Current Challenges, Alessio Cantore presents the results of his research on the use of AAV-derived vectors for delivery of FVIII and FIX in animal models, in terms of dose-response, biodistribution, and observed toxicities.