Sequence: TFR1 ligand peptide – Cys-(D-R9)-Cys (exact ligand sequence not disclosed)
antisense oligonucleotide (AON)
| Experiment Id | EXP000633 |
|---|---|
| Paper | Erythroid-Progenitor-Targeted Gene Therapy Using Bifunctional TFR1 Ligand-Peptides in Human Erythrop |
| Peptide | P1-9R |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | yes |
| Peptide Concentration | Charge ratio 5:1 (peptide:AON) |
| Rna Concentration | 428 nM |
| Mixing Ratio | 5:1 |
| Formulation Format | CPP–AON nanocomplex |
| Formulation Components | P1-9R + AON-V1 |
| Size Nm | 200.00 |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | LBCLs; UT7-EPO; primary CD34+ erythroid progenitors |
| Animal Model | |
| Administration Route | |
| Output Type | splice correction / gene expression rescue |
| Output Value | Normalization of WT FECH mRNA and protein; ↓PPIX |
| Output Units | |
| Output Notes | Efficient TFR1-mediated uptake and sustained splice correction |
| Toxicity Notes | No detectable cytotoxicity |
| Curation Notes |