Sequence: N-YArVRRrGPRGYArVRRrGPRr-C
PMO (splice-switching antisense morpholino)
| Experiment Id | EXP002436 |
|---|---|
| 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 (fluorescently tagged in some experiments) |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | Mild SMA model (F0): Smn−/−; SMN2+/+ with 4 SMN2 copies (JAX 005058); type III-like |
| Administration Route | subcutaneous (postnatal day 5) |
| Output Type | CNS delivery / nuclear localization; in vivo splice correction |
| Output Value | Nuclear localization in brain/spinal cord; increased FL-SMN2 in CNS & peripheral tissues |
| Output Units | |
| Output Notes | SC DG9-PMO injected at PD0 or PD5; assessed at PD7/PD13; ELISA shows higher CNS uptake vs unconjugated PMO; RT-qPCR shows increased FL-SMN2. |
| 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 |