1:1 cocktail of single Pip6a-PMOs (Dmd + Acvr2b) (in vivo)
Sequence: RXRRBRRXRYQFLIRXRBRXRB
PMO (phosphorodiamidate morpholino oligomer; splice-switching oligonucleotide)
| Experiment Id | EXP001991 |
|---|---|
| Paper | Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse |
| Peptide | 1:1 cocktail of single Pip6a-PMOs (Dmd + Acvr2b) (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 | Total 0.5 nmol PMO-conjugate mixture per TA (1:1). |
| Rna Concentration | Two separate PMO conjugates co-delivered. |
| Mixing Ratio | Mixture of two individual P-PMOs at 1:1 molar ratio (Pip6a-PMO(Dmd) + Pip6a-PMO(Acvr2b)). |
| Formulation Format | Covalent CPP–PMO conjugate(s) for in vivo splice switching in muscle (intramuscular administration). |
| Formulation Components | Separate single conjugates mixed prior to injection. |
| 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 | Used as comparator; bi-specific constructs show no significant loss of in vivo efficacy versus cocktail. |
| Toxicity Notes | |
| Curation Notes |