Sequence: N-YArVRRrGPRGYArVRRrGPRr-C
PMO (splice-switching antisense morpholino)
| Experiment Id | EXP002435 |
|---|---|
| Paper | DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous admin |
| Peptide | DG9 |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | |
| Mixing Ratio | |
| Formulation Format | covalent peptide–PMO conjugate |
| Formulation Components | DG9–PMO (ISS-N1 targeting PMO) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | Severe SMA Taiwanese model: Smn−/−; SMN2Tg/− (Cg-Tg(SMN2)2HungSmn1tm1Hung/J-derived) |
| Administration Route | subcutaneous (postnatal day 0) |
| Output Type | phenotypic rescue / survival; in vivo splice correction |
| Output Value | Median survival: 58 d (40 mg/kg); 121 d (80 mg/kg) |
| Output Units | |
| Output Notes | Single SC dose at PD0; improved weight, motor tests, breathing; increased FL-SMN2 and SMN protein in CNS and peripheral tissues. |
| Toxicity Notes | No apparent toxicity reported up to 80 mg/kg (serum markers, liver/kidney histology, urinary KIM-1); no increase in CD68+ macrophages vs NT. |
| Curation Notes |