BLOC1S1 depletion via splice-switching oligonucleotides improves mitochondrial respiration and rescues ALS phenotypes.
通过剪接转换寡核苷酸耗竭 BLOC1S1 可改善线粒体呼吸并挽救 ALS 表型。
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.12.026
Hor Jin-Hui, Ow Jin Rong, Ng Winanto, Ramasamy Boominathan, Tabaglio Tommaso, Lim Kimberly Ngee Hui, Bin Muzakar Muhammad Ikhsan, Lim Valerie Jing Wen, Gurrampati Rajasekhar Reddy, Rajarethinam Ravisankar, Ngo Shyuan T, Ramadass Venkataramanan, Ling Shuo-Chien, Lakshmanan Manikandan, Wee Keng Boon, Ng Shi-Yan