Sequence: Ac-RRRRRRRRR-NH2
ASO (antisense oligonucleotide; steric-blocking)
| Experiment Id | EXP001727 |
|---|---|
| Paper | The nuclear concentration required for antisense oligonucleotide activity in myotonic dystrophy cell |
| Peptide | R9 (nona-arginine) |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | no |
| Endosomal Escape Evidence | no |
| Peptide Concentration | At highest tested ASO dose (1 µM), peptide-only control corresponds to 10 µM R9; polyplex peptide concentration scales with ASO (10:1 molar). |
| Rna Concentration | ASO tested 0.1–1.0 µM (24 h incubation) for splice correction; imaging/uptake typically 0.1 µM Cy5-ASO. |
| Mixing Ratio | N/P = 6 (R9), corresponding to peptide:ASO molar ratio 10:1; polyplexes stabilized ~1 h RT. |
| Formulation Format | Noncovalent CPP/ASO polyplex (charge-driven nanoparticle) |
| Formulation Components | R9 + steric-blocking ASO (CAG5) in serum-free medium; noncovalent polyplexes. |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | Immortalized human DM1 myoblasts (DM11 cl5) and unaffected myoblasts (C25); fibroblast validation in some experiments |
| Animal Model | |
| Administration Route | |
| Output Type | in vitro splice correction (DM1 biomarkers) |
| Output Value | No measurable splice correction of MBNL1 (exons 5/7) or DMD (exon 78) vs untreated DM1 cells across tested doses. |
| Output Units | |
| Output Notes | 24 h treatment + 24 h recovery; RT-PCR quantification of exon inclusion. Confocal/FLIM indicate mostly vesicular/mitochondrial Cy5 signal with degradation; no homogeneous nuclear ASO distribution observed. |
| Toxicity Notes | Not the focus for R9; higher PF14 doses showed reduced viability; R9 peptide-only control included. |
| Curation Notes |