Sequence: (RXRRBR)(RXRRBR)XB
AMO (antisense morpholino oligonucleotide)
| Experiment Id | EXP001689 |
|---|---|
| Paper | Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing corr |
| Peptide | (RXRRBR)2XB |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.5–10 µM CPP-AMO (typical 1–5 µM) |
| Rna Concentration | Same as CPP-AMO (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_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | A-T lymphoblastoid cell line (AT203LA; heterozygous IVS28-159A>G) |
| Animal Model | |
| Administration Route | |
| Output Type | splice correction + protein/kinase restoration |
| Output Value | Strong splice correction and ATM protein restoration (~50% of WT by ELISA at effective doses); ATM kinase activity restored; radiosensitivity corrected. |
| Output Units | |
| Output Notes | CPP-AMO effective at ≥0.5–2.5 µM; demonstrates gene-dose effect (heterozygous vs homozygous). |
| Toxicity Notes | Not explicitly quantified. |
| Curation Notes |