Incorporation of a pH-Sensitive Linker into Lipid-ASO Conjugates Drives Increased Endosomal Membrane Remodeling Events.
Oligonucleotide therapeutics have great potential for the treatment of previously "undruggable" human diseases; however, their broad utility is limited by challenges in delivery and achieving sufficient endosomal escape. Systemic delivery of antisense oligonucleotides (ASOs) results in their primary accumulation in the...