Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteins
通过位点特异性地用烷基膦酸酯键取代硫代磷酸酯键,可以调节间隙型反义寡核苷酸(ASO)与细胞蛋白的相互作用,从而增强其治疗效果。
期刊:Nucleic Acids Research
影响因子:13.1
doi:10.1093/nar/gkz247
Migawa, Michael T; Shen, Wen; Wan, W Brad; Vasquez, Guillermo; Oestergaard, Michael E; Low, Audrey; De Hoyos, Cheryl L; Gupta, Ruchi; Murray, Susan; Tanowitz, Michael; Bell, Melanie; Nichols, Joshua G; Gaus, Hans; Liang, Xue-Hai; Swayze, Eric E; Crooke, Stanley T; Seth, Punit P