Nature Neuroscience, Published online: 12 December 2024; doi:10.1038/s41593-024-01838-6Duplication 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.
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This post is Copyright: Dong Yang | December 12, 2024
Nature Neuroscience