Table 1.
MOE ASOs Used in the Two-Step Intron Walks
| ASO Walk | ASO # | ASO Sequence | Target |
|---|---|---|---|
| Initial Walk in Intron 6a | |||
| 15–01 | 5′-CTGTAAGGAAAATAA-3′ | −1 to −15 | |
| 20–06 | 5′-AGGAAAATAAAGGAA-3′ | −6 to −20 | |
| 25–11 | 5′-AATAAAGGAAGTTAA-3′ | −11 to −25 | |
| 30–16 | 5′-AGGAAGTTAAAAAAA-3′ | −16 to −30 | |
| 35–21 | 5′-GTTAAAAAAAATAGC-3′ | −21 to −35 | |
| 40–26 | 5′-AAAAAATAGCTATAT-3′ | −26 to −40 | |
| 45–31 | 5′-ATAGCTATATAGATA-3′ | −31 to −45 | |
| 50–36 | 5′-TATATAGATATAGAT-3′ | −36 to −50 | |
| 55–41 | 5′-AGATATAGATAGCTA-3′ | −41 to −55 | |
| 60–46 | 5′-TAGATAGCTATATAT-3′ | −46 to −60 | |
| Initial Walk in Intron 7a | |||
| 01–15 | 5′-TGCTGGCAGACTTAC-3′ | 1 to 15 | |
| 06–20 | 5′-CATAATGCTGGCAGA-3′ | 6 to 20 | |
| 11–25 | 5′-ACTTTCATAATGCTG-3′ | 11 to 25 | |
| 16–30 | 5′-GATTCACTTTCATAA-3′ | 16 to 30 | |
| 21–35 | 5′-AGTAAGATTCACTTT-3′ | 21 to 35 | |
| 26–40 | 5′-ACAAAAGTAAGATTC-3′ | 26 to 40 | |
| 31–45 | 5′-GTTTTACAAAAGTAA-3′ | 31 to 45 | |
| 36–50 | 5′-ATAAAGTTTTACAAA-3′ | 36 to 50 | |
| 41–55 | 5′-AAACCATAAAGTTTT-3′ | 41 to 55 | |
| 46–60 | 5′-TCCACAAACCATAAA-3′ | 46 to 60 | |
| Microwalk in Intron 6a | |||
| 51–37 | 5′-ATATAGATATAGATA-3′ | −37 to −51 | |
| 52–38 | 5′-TATAGATATAGATAG-3′ | −38 to −52 | |
| 53–39 | 5′-ATAGATATAGATAGC-3′ | −39 to −53 | |
| 54–40 | 5′-TAGATATAGATAGCT-3′ | −40 to −54 | |
| 56–42 | 5′-GATATAGATAGCTAT-3′ | −42 to −56 | |
| 57–43 | 5′-ATATAGATAGCTATA-3′ | −43 to −57 | |
| 58–44 | 5′-TATAGATAGCTATAT-3′ | −44 to −58 | |
| 59–45 | 5′-ATAGATAGCTATATA-3′ | −45 to −59 | |
| 48–37 | 5′-ATATAGATATAG-3′ | −37 to −48 | |
| 49–38 | 5′-TATAGATATAGA-3′ | −38 to −49 | |
| 50–39 | 5′-ATAGATATAGAT-3′ | −39 to −50 | |
| 51–40 | 5′-TAGATATAGATA-3′ | −40 to −51 | |
| 52–41 | 5′-AGATATAGATAG-3′ | −41 to −52 | |
| 53–42 | 5′-GATATAGATAGC-3′ | −42 to −53 | |
| 54–43 | 5′-ATATAGATAGCT-3′ | −43 to −54 | |
| 55–44 | 5′-TATAGATAGCTA-3′ | −44 to −55 | |
| 56–45 | 5′-ATAGATAGCTAT-3′ | −45 to −56 | |
| Microwalk in Intron 7a | |||
| 08–25 | 5′-ACTTTCATAATGCTGGCA-3′ | 8 to 25 | |
| 09–26 | 5′-CACTTTCATAATGCTGGC-3′ | 9 to 26 | |
| 10–27 | 5′-TCACTTTCATAATGCTGG-3′ | 10 to 27 | |
| 11–28 | 5′-TTCACTTTCATAATGCTG-3′ | 11 to 28 | |
| 15–29 | 5′-ATTCACTTTCATAAT-3′ | 15 to 29 | |
| 14–28 | 5′-TTCACTTTCATAATG-3′ | 14 to 28 | |
| 13–27 | 5′-TCACTTTCATAATGC-3′ | 13 to 27 | |
| 12–26 | 5′-CACTTTCATAATGCT-3′ | 12 to 26 | |
| 10–24 | 5′-CTTTCATAATGCTGG-3′ | 10 to 24 | |
| 09–23 | 5′-TTTCATAATGCTGGC-3′ | 9 to 23 | |
| 08–22 | 5′-TTCATAATGCTGGCA-3′ | 8 to 22 | |
| 07–21 | 5′-TCATAATGCTGGCAG-3′ | 7 to 21 | |
| 18–29 | 5′-ATTCACTTTCAT-3′ | 18 to 29 | |
| 17–28 | 5′-TTCACTTTCATA-3′ | 17 to 28 | |
| 16–27 | 5′-TCACTTTCATAA-3′ | 16 to 27 | |
| 15–26 | 5′-CACTTTCATAAT-3′ | 15 to 26 | |
| 14–25 | 5′-ACTTTCATAATG-3′ | 14 to 25 | |
| 13–24 | 5′-CTTTCATAATGC-3′ | 13 to 24 | |
| 12–23 | 5′-TTTCATAATGCT-3′ | 12 to 23 | |
| 11–22 | 5′-TTCATAATGCTG-3′ | 11 to 22 | |
| 10–21 | 5′-TCATAATGCTGG-3′ | 10 to 21 | |
| Control Oligonucleotideb | |||
| 00–00 | 5′-TGCATCTCATTGTAG-3′ | None | |
a
Each ASO designation corresponds to the 5′ and 3′ nucleotide numbers of the intron sense sequence to which the ASO is complementary.
b
The sequence of the control ASO is unrelated to SMN2 exon 7 or to introns 6 and 7.