Sequence: (RXRRBR)(RXRRBR)XB
AMO (antisense morpholino oligonucleotide)
| Experiment Id | EXP001690 |
|---|---|
| Paper | Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing corr |
| Peptide | (RXRRBR)2XB |
| Delivery Success Class | no |
| In Vivo Flag | yes |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | Dose: 60 mg/kg/day (CPP-AMO conjugate) single or 4 daily injections |
| Rna Concentration | Same as dose (covalent conjugate) |
| Mixing Ratio | Covalent CPP–AMO conjugate (1:1 peptide:AMO) |
| Formulation Format | CPP-conjugated AMO (AVI Biopharma peptide–morpholino conjugate) |
| Formulation Components | (RXRRBR)2XB peptide covalently conjugated to AMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | C57BL/6 mice (wild-type); brain distribution study |
| Administration Route | Intravenous (tail vein) |
| Output Type | in vivo uptake / biodistribution |
| Output Value | FITC signal detected throughout brain including cerebellum; Purkinje cell layer strongly stained; nuclear localization observed in Purkinje cells; signal increased with 4 daily injections. |
| Output Units | |
| Output Notes | Single IV injection vs 4 consecutive daily injections at 60 mg/kg/day; analysis 24 h after last injection; anti-FITC immunostaining and confocal microscopy used. |
| Toxicity Notes | No apparent signs of toxicity or behavioral changes; H&E staining showed no obvious brain morphology changes (as reported). |
| Curation Notes |