C12-r8 (lauric acid–octaarginine)
Sequence: C12-r8 (D-type octaarginine; lauric acid coupled to N-terminus of D-octaarginine)
| Experiment Id | EXP001809 |
|---|---|
| Paper | Nose-to-brain delivery of enveloped RNA - cell permeating peptide nanocomplexes for the treatment of |
| Peptide | C12-r8 (lauric acid–octaarginine) |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | Dose: 6.5 nmol (single-dose biodistribution) or 15 nmol total (efficacy; 3×5 nmol) |
| Mixing Ratio | N/P (C12-r8:RNA) = 4:1 |
| Formulation Format | Enveloped nanocomplex (ENCP): core C12-r8/miRNA nanocomplex, PEG-PGA shell; intranasal nose-to-brain delivery |
| Formulation Components | PEG-PGA ENCPs with miR-132 mimic at N/P 4:1; control: scrambled miRNA ENCPs | Enveloping polymer: PEG-PGA (used for in vivo studies) |
| Size Nm | 96.00 |
| Zeta Mv | 4.00 |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | APPNL−G-F knock-in Alzheimer’s disease mouse model (11-month old males) |
| Administration Route | Intranasal (4 µL per nostril with 2 min intervals); single dose 6.5 nmol for biodistribution; 3 doses of 5 nmol (2 h intervals) for efficacy |
| Output Type | In vivo RNA delivery + functional effect (qPCR in brain regions; mRNA target modulation) |
| Output Value | Hippocampus miR-132 significantly increased at 24 h (p=0.01); GATA2 mRNA ↓ ~10% (olfactory bulb) and Rb1 mRNA ↑ ~20% (hippocampus) vs control |
| Output Units | |
| Output Notes | Demonstrates nose-to-brain delivery and RNA activity on downstream targets. |
| Toxicity Notes | No detailed systemic toxicity endpoints reported in main text. |
| Curation Notes |