Nanodiscoidal Nucleic Acids for Gene Regulation (2023)
22A ApoA1 mimetic peptide A / lipid-ASO ND1
Sequence: PVLDLFRELLNELLEALKQKLK
| Experiment Id | EXP002481 |
|---|---|
| Paper | Nanodiscoidal Nucleic Acids for Gene Regulation |
| Peptide | 22A ApoA1 mimetic peptide A / lipid-ASO ND1 |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | medium |
| In Vitro Functional Effect | yes |
| 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 + ApoA1 mimetic peptide A + EZN2968 ASO |
| Size Nm | 13.00 |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | HeLa cells |
| Animal Model | |
| Administration Route | |
| Output Type | HIF-1α mRNA knockdown and cell viability |
| Output Value | Reduced HIF-1α transcript vs control; less active than NNA3 |
| Output Units | |
| Output Notes | HeLa screen of ASO-ND/NNA scaffolds; ASO-ND 1 uses peptide A with lipid-linked ASO. Functional knockdown shown by RT-qPCR and cell viability assay. |
| Toxicity Notes | No major toxicity emphasized for NNA scaffold; cell viability reduction relates to HIF-1α knockdown in cancer cells. |
| Curation Notes |