In vivo repair of a protein underlying a neurological disorder by programmable RNA editing
John R. Sinnamon, Susan Y. Kim, Jenna R. Fisk, Zhen Song, Hiroyuki Nakai, Sophia Jeng, Shannon K. McWeeney, Gail Mandel
This study uses RNA base editing to repair a mutation in a mouse model of Rett syndrome, restoring MeCP2 function in hippocampal neurons.
Cell Reports · Jul 1, 2020Peer-reviewedNeuroscience