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 10 months 4 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.

Submit comment | Blog It | Email It | Keywords: gene therapy gene delivery gene cassettes hemiphilia gene therapy codon optimation scAAV vectors