Table 1. Gapmer ASO sequences and their on-target mRNA transcripts.
Isis No | Alternative ID | Sequence | Modification | Gene | Genbank number | On-target Species | First Toxic Dose (mg/kg) >1000 IU/ml ALT (96 h) |
---|---|---|---|---|---|---|---|
569713 | ASO ctrl | 5′-GACGCGCCTGAAGGTT-3′ | LNA | N/A, control | N/A | Mouse | >300 |
571035 | FVII-1 | 5′-CAGATATAGGACTGGA-3′ | LNA | F7 | NM_010172.3 | Human | >300 |
571033 | FXI-3 | 5′-ATCCAGAGATGCCTCC-3′ | LNA | F11 | NM_028066.1 | Mouse | >300 |
569714 | FXI-4 | 5′-GGCCACCACGCTGTCA-3′ | LNA | F11 | NM_028066.1 | Mouse | >300 |
571034 | FXI-5 | 5′-TGCCACCGTAGACACG-3′ | LNA | F11 | NM_028066.1 | Mouse | >300 |
569715 | SOD1–1 | 5′-GGACACATTGGCCACA-3′ | LNA | Sod1 | NM_011434.1 | Mouse | >300 |
569716 | FVII-2 | 5′-CCCTGGTGTACACCCC-3′ | LNA | F7 | NM_010172.3 | Mouse | 300 |
569717 | PTEN | 5′-ATCATGGCTGCAGCTT-3′ | LNA | Pten | NM_008960.2 | Mouse | 33 |
569718 | FVII-3 | 5′-TGGTCCCTGCAGTACT-3′ | LNA | F7 | NM_010172.3 | Mouse | 100 |
569719 | FXI-1 | 5′-GTCTGTGCATCTCTCC-3′ | LNA | F11 | NM_028066.1 | Mouse | 11 |
443919 | FXI-1-M | 5′-GTCTGTGCATCTCTCC-3′ | 2’ MOE | F11 | NM_028066.1 | Mouse | 300 |
464917 | FXI-1-C | 5′-GTCTGTGCATCTCTCC-3′ | cEt | F11 | NM_028066.1 | Mouse | 11 |
569720 | FXI-2 | 5′-GTCAGTATCCCAGTGT-3′ | LNA | F11 | NM_028066.1 | Mouse | 100 |
444011 | FXI-2-M | 5′-GTCAGTATCCCAGTGT-3′ | 2’ MOE | F11 | NM_028066.1 | Mouse | 300 |
465178 | FXI-2-C | 5′-GTCAGTATCCCAGTGT-3′ | cEt | F11 | NM_028066.1 | Mouse | 100 |
569721 | SOD1–2 | 5′-TGAGGTCCTGCACTGG-3′ | LNA | Sod1 | NM_011434.1 | Mouse | 33 |
529933 | SOD1–2-M | 5′-TGAGGTCCTGCACTGG-3′ | 2’ MOE | Sod1 | NM_011434.1 | Mouse | 300 |
508031 | SOD1–2-C | 5′-TGAGGTCCTGCACTGG-3′ | cEt | Sod1 | NM_011434.1 | Mouse | 33 |
554219 | BIRC5 | 5′- CTCAATCCATGGCAGC-3′ | LNA | Birc5 | NM_001168.2 | Human | 300 |
The 16-mer antisense oligonucleotides were synthesized with the indicated modifications (underlined sequences). The center (gap) of the antisense oligonucleotides (not underlined) consist of deoxynucleotide bases and phosphorothioate backbone linkages.