NeurosciencebioRxivPreprint
Counter-balancing X-linked Mecp2 hypofunction by hyperfunction ameliorates disease features in a model of Rett syndrome: implications for genetic therapies
Christopher Michael McGraw, Sirena Soriano, Shuang Hao…·Rodney C. Samaco
In a mouse model of Rett syndrome, counterbalancing mosaic LOF with complementary mosaic GOF improves phenotypic outcome.
DOI 10.1101/2024.01.18.576265