Article
Hyperfunctional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice.
Blood - 29 Nov 2012
Cantore Alessio, Nair Nisha, Della Valle Patrizia, Di Matteo Mario, Màtrai Janka, Sanvito Francesca, Brombin Chiara, Di Serio Clelia, D'Angelo Armando, Chuah Marinee, Naldini Luigi, Vandendriessche Thierry
Abstract excerpt
Gene therapy may provide a cure for hemophilia and overcome the limitations of protein replacement therapy. Increasing the potency of gene transfer vectors may allow improvement of their therapeutic index, as lower doses can be administered to achieve therapeutic benefit, reducing toxicity of in vivo administration. Here we generated codon-usage optimized and hyperfunctional factor IX (FIX) transgenes carrying an...
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