Cryptic Exon Silencing Restoration

**Molecular Mechanism and Rationale** The TAR DNA-binding protein 43 (TDP-43), encoded by the TARDBP gene, serves as a critical RNA-binding protein (RBP) that orchestrates complex post-transcriptional regulatory networks essential for neuronal homeostasis. Under physiological conditions, TDP-43 functions as a master regulator of cryptic exon silencing through its preferential binding to UG-rich and GU-rich sequences located within introns and 3' untranslated regions of target transcripts. The p...