Table 4.
Oral formulations | Tissues | Exon 23 skipping % |
IHC | WB | |
---|---|---|---|---|---|
Group I No alginate (n = 6) |
ZM2 (2.5 mg)-AON (225 μg) (n = 3) |
Diaphragm | 0 | − | − |
Intestine | 0 | − | − | ||
Gastrocnemius | 0 | − | − | ||
Quadriceps | 0 | − | − | ||
Heart | 0 | − | − | ||
ZM2 (2.5 mg) (n = 3) |
Diaphragm | 0 | − | − | |
Intestine | 0 | − | − | ||
Gastrocnemius | 0 | − | − | ||
Quadriceps | 0 | − | − | ||
Heart | 0 | − | − | ||
| |||||
Group II +alginate (n = 6) |
ZM2 (2.5 mg)-AON (200 μg) (n = 3) |
Diaphragm | 8 | + | − |
Intestine | 0 | + | + | ||
Gastrocnemius | 0 | − | − | ||
Quadriceps | 0 | − | − | ||
Heart | 0 | − | − | ||
ZM2 (2.5 mg) (n = 3) |
Diaphragm | 0 | − | − | |
Intestine | 0 | − | − | ||
Gastrocnemius | 0 | − | − | ||
Quadriceps | 0 | − | − | ||
Heart | 0 | − | − | ||
| |||||
Group III Untreated (n = 3) |
— | Diaphragm | 0 | − | − |
Intestine | 0 | − | − | ||
Gastrocnemius | 0 | − | − | ||
Quadriceps | 0 | − | − | ||
Heart | 0 | − | − |