Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose

davidd submitted, created time 9 months 2 weeks (www.nature.com)

The article represents adeno-associated virus vectors optimiztion for potential application in future hemophilia B clinical trials and liver-directed gene delivery.
These studies underscore the need for rigorous analysis and optimization of vector genome cassettes.

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