Efficient precise in vivo base editing in adult dystrophic mice
L. Xu, C. Zhang, H. Li, P. Wang, Y. Gao, P. J. Mohler, N. Mokadam, J. Ma, W. D. Arnold, R. Han
This paper improves an adenine base editor for in vivo use, achieving efficient dystrophin restoration in dystrophic mice with minimal off-target effects.
bioRxiv · Jun 24, 2020Bioengineering