Compositions and methods for treating tdp-43 proteinopathy

Published: 06/10/2025

20. A method of reducing phosphorylated TAR-DNA binding protein-43 (TDP-43) in a cell comprising administering a UNC13A cryptic exon splice variant specific inhibitor, wherein: (a) the UNC13A cryptic exon splice variant comprises a cryptic exon between exon 20 and exon 21 of the UNC13A cryptic exon …