[EN] POLYNUCLEOTIDES HAVING BIOREVERSIBLE GROUPS<br/>[FR] POLYNUCLÉOTIDES POSSÉDANT DES GROUPES BIORÉVERSIBLES
申请人:UNIV CALIFORNIA
公开号:WO2014031575A1
公开(公告)日:2014-02-27
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).