Dual-targeting snRNA gene therapy rescues STMN2 and UNC13A splicing in TDP-43 proteinopathies
双靶向snRNA基因疗法可挽救TDP-43蛋白病中的STMN2和UNC13A剪接
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doi:10.64898/2025.12.01.691001
Gomberg, Trent A; Elmsaouri, Sara; Kopalle, Hema M; Baughn, Michael W; Beccari, Melinda S; McAlonis-Downes, Melissa; Artates, Jonathan W; Pant, Deepak; Mak, Harriet; Smargon, Aaron A; Sander, Tynan C; Garcia, Esaul; Lee, Dominic P; Cleveland, Don W; Yeo, Gene W