Table 4.
Summary of results of dystrophin restoration studies.
| 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 | − | − | ||