Table 1.
Sequences and binding coordinates of exon skipping AOs designed for the human SMN and mouse Smn transcripts. The AO length and melting temperature (Tm) of the 2′-O-methyl AO sequence is listed.
| Species and Exon | AO Name/Number | Coordinates | Sequence 5′-3′ | Length (bp) | Tm (ᵒC) |
|---|---|---|---|---|---|
| Human Exon 7 |
h1 h2 h3 h4 h5 h6 h7 |
SMN H7A(− 08 + 17) SMN H7A(+ 01 + 25) 1 SMN H7A(+ 07 + 31) 1 SMN H7A(+ 06 + 27) 1 SMN H7A(+ 13 + 32) 1 SMN H7A(+ 22 + 46) 1 SMN H7D(+ 17 − 13) |
UGA UUU UGU CUA AAA CCC UGU AAG G CUU CUU UUU GAU UUU GUC UAA AAC C ACC UUC CUU CUU UUU GAU UUU GUC U UCC UUC UUU UUG AUU UUG UCU G CAC CUU CCU UCU UUU UGA UU UUA AGG AAU GUG AGC ACC UUC CUU C CUG GCA GAC UUA CUC CUU AAU UUA AGG AAU |
25 25 25 22 20 25 30 |
66.4 58.6 64.7 59.5 59.2 70.5 70.2 |
| Human Exon 5 |
h8 h9 |
SMN H5A(+ 04 + 28) SMN H5A(+ 29 + 53) |
GUG GUG GGC CAU UGA AUU UUA GAC C UGG GGU GGU GGU GGU GGC GGU GGC G |
25 25 |
71.1 87.0 |
| Mouse Exon 7 |
m1 m2 m3 m4 m5 m6 m7 m8 |
Smn M7A(+ 02 + 31) Smn M7A(+ 06 + 27) Smn M7A(+ 07 + 31) Smn M7A(+ 07 + 36) 2 Smn M7A(+ 13 + 32) Smn M7D(+ 11 − 14) Smn M7D(+ 11 − 19) Smn M7D(+ 17 − 13) |
ACU UUC CUU CUU UUU UAU UUU GUC UGA AAC UCC UUC UUU UUU AUU UUG UCU G ACU UUC CUU CUU UUU UAU UUU GUC U UGA GCA CUU UCC UUC UUU UUU AUU UUG UCU CAC UUU CCU UCU UUU UUA UU AAU GAC AGA CUU ACU UCU UAA UUU G UUU AAA AUG ACA GAC UUA CUU CUU AAU UUG AUG ACA GAC UUA CUU CUU AAU UUG UAU GUG |
30 22 25 30 20 25 30 30 |
63.5 56.9 59.8 67.2 52.6 60.7 61.9 65.9 |
| Mouse Exon 5 |
m9 m10 m11 m12 m13 |
Smn M5A(− 06 + 19) Smn M5A(+ 12 + 36) Smn M5A(+ 32 + 54) Smn M5A(+ 56 + 77) Smn M5A(+ 18 − 07) |
CGU UGA AUU UUA GAC CUG GCU AUA A AGG CGG CGG CGG CGG GCC GUU GAA U AAG GGG GGA GGG GGU AGU GGA GGC G AAC GGG GGC AUC CAG CAC GGC AG UAC UUA CUG GUG GUC CUG AAG GGA A |
25 25 25 23 25 |
65.8 86.8 85.9 81.6 75.7 |