Figure 2. Summary of AGT ASO development and research in animal models from 1993 to 2017.

‘MOE Gapmer’ represents that ASO is flanked by MOE residues, which increases ASO binding affinity and RNase H cleavage at the center region of ASO. ‘cEt Gapmer’ represents that the ASO has 10–14 nucleotides central DNA region flanked by two to five of cEt residues, which improves hybridization with complementary sequences. Related representative references for each disease are denoted.