Disruption of splicing-regulatory elements using CRISPR/Cas9 to rescue spinal muscular atrophy in human iPSCs and mice.

2020 Li, J., Lin, X., Tang, C., Lu, Y., Hu, X., Zuo, E., Li, H., Ying, W., Sun, Y., Lai, L., Chen, H., Guo, X., Zhang, Q., Wu, S., Zhou, C., Shen, X., Wang, Q., Lin, M., Ma, L., Wang, N., Krainer A.R., Shi, L.*, Yang, H.* & Chen W.*