Translatable electrophysiological and behavioral abnormalities in a humanized model of SYNGAP1-disorder
Alex J. Félix, Brandon L. Brown, Nicolas Marotta, Maximilian J. Gessner, Mika Houserova, Icnelia Huerta-Ocampo, Taryn Wilson, Rani Randell, Jennine M. Dawicki-McKenna, Dulce Reinhardt, Keita Uchida, Ian McSalley, Jillian L. McKee, Ingo Helbig, Michael J. Boland, , Benjamin L. Prosser
The paper develops humanized mouse models for SYNGAP1 and STXBP1 disorders, showing similar electrophysiological and behavioral abnormalities in mice and patients, and demonstrates the efficacy of antisense oligonucleotides in modulating SYNGAP1 expression.
Molecular Psychiatry · Jul 14, 2026Peer-reviewedNeuroscience