Maturation-dependent splicing alterations constrain SYNGAP1 splice-switching therapy
Juliette A. Kamp, Kim A Wijnant, Nadine Maas, Deniz Gülyurt, Melissa JM Rieder, Mehrnoush Aghadavoud Jolfaei, Cristina Gontan, Steven A. Kushner, Ype Elgersma, Lisenka E.L.M. Vissers, Nael Nadif Kasri, Femke M. S. de Vrij
The paper shows that SYNGAP1 splice-switching therapy may be ineffective in mature neurons due to reduced A3SS transcript abundance.
bioRxiv · Aug 31, 2026 · PreprintNeuroscience