The disclosure provides methods and compositions for delivering polynucleotides into cells. The disclosure provides transiently protected polynucleotides comprising an anionic charge-neutralizing moiety/group, which may also confer additional functionality. These compounds can enter the cytosol of cells by endocytic or macropinocytic mechanisms. The transient protecting group is bioreversible, i.e., once inside a cell, it is designed to be removed by enzymatic activity or by passive intracellular methods (e.g., changes in pH or reductive environment).
该披露提供了将多核苷酸传递到细胞中的方法和组合物。该披露提供了暂时保护的多核苷酸,其中包含一个阴离子电荷中和的基团,这可能还赋予了额外的功能。这些化合物可以通过内吞或巨胞吞噬机制进入细胞的细胞质。暂时的保护基团是可
生物逆转的,即一旦进入细胞内,它被设计为能够通过酶活性或被动的细胞内方法(例如,pH值的变化或还原环境)来去除。