Loss of TDP-43 induces synaptic dysfunction that is rescued by UNC13A splice-switching ASOs
Matthew J. Keuss, Peter Harley, Eugeni M. Ryadnov, Rachel E. Jackson, Matteo Zanovello, Oscar G. Wilkins, Simone Barattucci, Puja R. Mehta, Marcio Guiomar Oliveira, Joanna E. Parkes, Aparna Sinha, Andrés F. Correa-Sánchez, Peter L. Oliver, Elizabeth Fisher, Giampietro Schiavo, , Pietro Fratta
This study shows that loss of TDP-43 causes synaptic dysfunction through a cryptic exon in UNC13A, which is rescued by antisense oligonucleotides.
bioRxiv · Jun 20, 2024 · PreprintNeuroscience