RNA干扰
基因沉默
小干扰RNA
寡核苷酸
体内
核酸
反式siRNA
计算生物学
锁核酸
核糖核酸
RNA沉默
生物
细胞生物学
分子生物学
基因
化学
遗传学
生物化学
作者
Ivan Zlatev,Adam Castoreno,Christopher R. Brown,June Qin,Scott Waldron,Mark K. Schlegel,Rohan Degaonkar,Svetlana Shulga‐Morskaya,Huilei Xu,Swati Gupta,Shigeo Matsuda,Akin Akinc,Kallanthottathil G. Rajeev,Muthiah Manoharan,Martin A. Maier,Vasant Jadhav
摘要
We report rapid, potent reversal of GalNAc-siRNA-mediated RNA interference (RNAi) activity in vivo with short, synthetic, high-affinity oligonucleotides complementary to the siRNA guide strand. We found that 9-mers with five locked nucleic acids (LNAs) have the highest potency across several targets. Our modular, sequence-specific approach, named REVERSIR, may enhance the therapeutic profile of any long-acting GalNAc-siRNA (short interfering RNA) conjugate by enabling control of RNAi pharmacology.
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(2025-6-4)
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