This work was supported by the National Natural Science Foundation of China (Nos. 31071171 and 81170532) and the National High Technology Research and Development Program of China (863 Program, No. 2012AA020810).
Hemophilia B is a hemorrhagic disease caused by the deficiency of clotting factor IX (FIX). Gene therapy might be the ultimate strategy for the disease. However, two main problems that should be solved in gene thera...