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. Author manuscript; available in PMC: 2019 Jul 1.
Published in final edited form as: Wiley Interdiscip Rev RNA. 2018 Apr 25;9(4):e1476. doi: 10.1002/wrna.1476

Table 1.

Cancer-associated human isoforms targeted by splice-switching ASO

Gene name ASO chemistry Type of splicing correction Tumor type Tested in cell lines Tested in vivo References
STAT3 PMO exon 23 skipping breast √ √ 424
MDM4 PMO exon 6 skipping skin, lymphoma √ √ 425
ERBB4 LNA exon 26 skipping breast √ √ 444
BCL2L1 2′-MOE/PS exon 2 skipping skin √ √ 239,240,445
GLDC 2′-MOE/PS exon 7 skipping lung √ √ 446
PKM2 2′-MOE/PS exon 9 inclusion brain √ n.d. 447
MCL1 PMO exon 2 skipping skin √ n.d. 271
MDM2 PNA exon 4 skipping uterine √ n.d. 448
BRCA2 2′-OMe/PS cryptic exon skipping breast √ n.d. 449
IL5R 2′-MOE/PS exon 5 skipping lymphoma √ n.d. 450
FGFR1 PMO exon α inclusion brain √ n.d. 451
MSTR1 PMO exon 11 skipping breast and stomach √ n.d. 452
USP5 PMO alternative 5′SS brain √ n.d. 453

PMO: phosphorodiamidate morpholino oligomer; LNA: locked nucleic acid; PNA: peptide nucleic acid; 2′-MOE/PS: 2′-O-(2-methoxyethyl)/phosphorothioate; 2′-OMe/PS: 2′ O-methyl/phosphorothioate; n.d.: not determined.