Amelioration of hemophilia B through CRISPR/Cas9 induced homology-independent targeted integration
X. Chen, X. Niu, Y. Liu, R. Zheng, L. Wang, L. Yang, J. Lu, S. Yin, Y. Shao, Y. Wei, J. Pan, A. Sayed, X. Ma, M. Liu, F. Jing, , D. Li
This study uses CRISPR/Cas9 to induce homology-independent targeted integration in a rat hemophilia B model, achieving therapeutic correction with 3.66% knock-in efficiency and normalization of clotting time.
bioRxiv · Mar 18, 2021 · PreprintBioengineering