Sequence: RXRRBRRXRYQFLIRXRBRXRB
PMO (phosphorodiamidate morpholino oligomer; splice-switching oligonucleotide)
| Experiment Id | EXP001990 |
|---|---|
| Paper | Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse |
| Peptide | Bi-specific D3 (in vivo) |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.5 nmol conjugate per TA. |
| Rna Concentration | Contains 2 PMOs per conjugate (total PMO equivalents delivered in the conjugate). |
| Mixing Ratio | Bi-specific conjugate (both PMOs on one Pip6a; click linkage for second PMO). |
| Formulation Format | Covalent CPP–PMO conjugate(s) for in vivo splice switching in muscle (intramuscular administration). |
| Formulation Components | D3 bi-specific Pip6a–PMO–PMO conjugate. |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | mdx mice (C57BL/10ScSn-Dmdmdx/J); tibialis anterior muscle intramuscular injection |
| Administration Route | Intramuscular injection into TA: 0.5 nmol peptide–PMO in 30 µL 0.9% saline; analysis 2 weeks post-injection |
| Output Type | In vivo functional RNA delivery: exon skipping in TA muscle (RT-PCR + qPCR) |
| Output Value | ~35–40% Dmd exon23 skipped transcripts by qPCR; Acvr2b exon5 skipping present (lower than Dmd). |
| Output Units | |
| Output Notes | Comparable in vivo splice-switching activity to D2 and to cocktail treatment. |
| Toxicity Notes | |
| Curation Notes |