Sequence: RRRRRRG
PMO (splice-switching antisense morpholino)
| Experiment Id | EXP002437 |
|---|---|
| Paper | DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous admin |
| Peptide | R6G |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | no |
| Label Confidence | medium |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | |
| Mixing Ratio | |
| Formulation Format | covalent peptide–PMO conjugate |
| Formulation Components | R6G–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 effect / survival (benchmark CPP–PMO) |
| Output Value | Median survival: 17 d (40 mg/kg) |
| Output Units | |
| Output Notes | Single SC dose at PD0; included as benchmark peptide–PMO comparator; reported less effective than DG9-PMO. |
| Toxicity Notes | |
| Curation Notes |