Sequence: (RXRRBR)(RXRRBR)XB
AMO (antisense morpholino oligonucleotide)
| Experiment Id | EXP001688 |
|---|---|
| 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 | yes |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | 0.5–10 µM CPP-AMO (typical 2.5–5 µM; 4-day treatment for protein assays; 48 h for mRNA assays) |
| 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 (TATC; homozygous c.7865C>T) |
| Animal Model | |
| Administration Route | |
| Output Type | splice correction + protein restoration |
| Output Value | ≥80% mutant transcript correction at ≥2 µM; ATM protein restored to near WT levels; ATM kinase activity restored (SMC1/KAP1 phosphorylation); radiosensitivity abrogated; ATM detectable up to ~20 days after single 5 µM dose. |
| Output Units | |
| Output Notes | Dose-response 0.5–10 µM; RT-PCR/RT-qPCR, western blot, ELISA, FACS (ATM Ser1981), clonogenic survival assays; prolonged intracellular fluorescence with AMCA tag indicates uptake/retention. |
| Toxicity Notes | No explicit cytotoxicity assay reported for LCLs in vitro; phenotypic rescue observed at 1–5 µM. |
| Curation Notes |