Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing
F. Hatanaka, K. Suzuki, K. Shojima, J. Yu, Y. Takahashi, A. Sakamoto, J. Prieto, M. Shokhirev, C. R. Esteban, E. Nunez-Delicado, J. C. I. Belmonte
The paper combines gene supplementation and genome editing to treat spinal muscular atrophy in mice, showing long-term therapeutic benefits.
bioRxiv · Apr 6, 2023Bioengineering