Nanodiscoidal Nucleic Acids for Gene Regulation (2023)
C-terminal GGC 22A ApoA1 mimetic peptide / NNA3
Sequence: PVLDLFRELLNELLEALKQKLGGC
| Experiment Id | EXP002490 |
|---|---|
| Paper | Nanodiscoidal Nucleic Acids for Gene Regulation |
| Peptide | C-terminal GGC 22A ApoA1 mimetic peptide / NNA3 |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | 100 nM ASO in most in vitro assays; 550 nM ASO in H1299 spheroids; 0.7 mg/kg ASO in vivo |
| Mixing Ratio | ~25-35 ASO copies per NNA depending scaffold; ND3 ~25-26 copies per nanodisc |
| Formulation Format | Nanodiscoidal nucleic acid / ASO-modified nanodisc |
| Formulation Components | DMPC + 10 mol% Ptd-thioethanol + C-terminal GGC ApoA1 mimetic peptide B + EZN2968 ASO on peptide belt and lipid faces |
| Size Nm | 13.00 |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | C57BL/6 mice; liver/kidney/spleen/lung/fat harvest 48 h after tail-vein injection |
| Administration Route | tail-vein intravenous injection |
| Output Type | HIF-1α mRNA knockdown in harvested organs |
| Output Value | 0.7 mg/kg ASO; significant kidney HIF-1α knockdown; liver showed knockdown trend |
| Output Units | |
| Output Notes | NNA3 localized mainly to liver and kidney; kidney HIF-1α knockdown was statistically significant. This is functional in vivo ASO delivery. |
| Toxicity Notes | No major toxicity emphasized for NNA scaffold; cell viability reduction relates to HIF-1α knockdown in cancer cells. |
| Curation Notes |