Duplication of the MECP2 gene (encoding methyl-CpG-binding protein 2) causes MECP2 duplication syndrome. Here the authors develop a Cas13Y system capable of targeting the MECP2 mRNA for degradation and reducing protein levels in the brain of humanized MECP2-transgenic mice and nonhuman primates.
- Dong Yang
- Xiaoqing Wu
- Hui Yang
