In vitro cellular phenotypes of cortical neurons from R255X MECP2 knock-in mice are improved by either expression of wildtype MeCP2 or read-through with G418
Xin Xu, Jonathan K. Merritt, Steven James Gray, Jeffrey Lorenz Neul, Lucas D. Pozzo‐Miller
The paper shows that expressing wildtype MeCP2 or using G418 treatment in R255X MECP2 knock-in neurons restores cellular phenotypes to wildtype levels.
bioRxiv · Feb 17, 2025 · PreprintNeuroscience