Short RNA chaperones promote aggregation-resistant TDP-43 conformers to mitigate neurodegeneration
Katie E. Copley, Jocelyn C. Mauna, Helen L. Danielson, Qizan Chen, Busra Ozguney, Marilyn Ngo, Longxin Xie, Ashleigh Smirnov, Matt Davis, Leland C. Mayne, Miriam Linsenmeier, Jack D. Rubien, Cristian A Bergmann, Bede Portz, Bo Lim Lee, , James Shorter
Short UG-rich RNAs bind and stabilize TDP-43 RNA-recognition motifs to allosterically destabilize its prion-like domain helical region, preventing aggregation and mitigating neurodegeneration in cells and mice.
Science · May 7, 2026Peer-reviewedBiochemistry✦ In-depth digest