Dual-targeting snRNA gene therapy rescues STMN2 and UNC13A splicing in TDP-43 proteinopathies
Trent A. Gomberg, Sara Elmsaouri, Hema Kopalle, Michael W. Baughn, Melinda S. Beccari, Melissa McAlonis‐Downes, Jonathan W. Artates, Deepak Pant, Harriet Mak, Aaron A. Smargon, Tynan C. Sander, Esaul Garcia, Dominic Paul Lee, Don W. Cleveland, Gene Wei-Ming Yeo
This study develops a gene therapy strategy using snRNAs to correct mis-splicing of STMN2 and UNC13A in TDP-43 proteinopathies, showing restoration of protein levels and axonal regeneration in motor neurons.
bioRxiv · Dec 3, 2025 · PreprintNeuroscience