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Monomeric TDP-43 binds GU-rich RNA via its NTD but only its oligomers block cryptic exon inclusion

Polymenidou, M.; Zbinden, A.; Smok, I.; Pellegrini, F.; Oertel, A.; Clery, A.; Wiersma, V. I.; Huang, W.-P.; Gatta, B.; Zhong, W.; Li, Y.; Götze, M.; Peter, M.; Bargenda, N.; Hruska-Plochan, M.; Caflisch, A.; Fratta, P.; Shelkovnikova, T.; Allain, F. H.- T.; Leitner, A.

2026-10-05 molecular biology
10.64898/2026.10.01.755875 bioRxiv
Show abstract

TDP-43 aggregation is a pathological feature of several neurodegenerative diseases, resulting in nuclear loss of function and RNA dysregulation, including cryptic exon derepression. TDP-43 comprises two RNA recognition motifs required for canonical RNA binding, a low-complexity domain implicated in phase separation, and an N-terminal domain (NTD) that mediates oligomerization. While NTD-driven oligomerization is critical for TDP-43 splicing function, some studies suggest a direct interaction of NTD with RNA. Here, we investigated RNA binding by the NTD and its functional interplay with oligomerization. We show that the NTD preferentially binds UG-rich RNA via residues located at the oligomerization interface. The NTD engaged RNA only on monomeric TDP-43, as RNA binding and oligomerization compete for the same interface and rely on similar interaction modes. By disentangling these competing activities, we demonstrated that oligomerization, but not NTD-RNA interaction, is essential for TDP-43-dependent alternative splicing and repression of cryptic exons. Our findings uncover a previously unappreciated RNA-binding activity within the TDP-43 NTD that directly competes with oligomerization and establish oligomerization as a key determinant of TDP-43 cryptic exon repression.

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