U7 small nuclear RNA splice-switching therapeutics for STMN2 and UNC13A in Amyotrophic Lateral Sclerosis.
U7 小核 RNA 剪接转换疗法用于治疗肌萎缩侧索硬化症中的 STMN2 和 UNC13A。
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doi:10.1101/2025.11.26.690143
Mehta Puja R, Solomon Tomas, Pickles Sarah, Harley Peter, Barioglio Michela, Schweingruber Christoph, Marrero-Gagliardi Alessandro, Gao Yujing, Mattedi Francesca, Barattucci Simone, Lin Lilian Tsai-Wei, Ryadnov Eugeni, Zanovello Matteo, Cammack Alexander J, Isaacs Adrian M, Burrone Juan, Shaw Christopher E, Keuss Matthew J, Petrucelli Leonard, Fratta Pietro, Ruepp Marc-David