POLYNUCLEOTIDES HAVING BIOREVERSIBLE GROUPS

摘要

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).