Induction of splice correction by cell-penetrating peptide nucleic acids (2006)
Sequence: CYGRKKRRQRRR-NH2
PNA (splice-switching antisense)
| Experiment Id | EXP000846 |
|---|---|
| Paper | Induction of splice correction by cell-penetrating peptide nucleic acids |
| Peptide | Tat |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | no |
| Endosomal Escape Evidence | yes |
| Peptide Concentration | 1–10 µM (dose range; often 2–10 µM) |
| Rna Concentration | Same as conjugate concentration (1:1 CPP–PNA conjugate) |
| Mixing Ratio | 1:1 (covalent conjugate) |
| Formulation Format | CPP–PNA disulfide conjugate |
| Formulation Components | Cell-penetrating peptide (CPP) disulfide-linked to splice-correcting PNA (M705). Endosomal escape enhancers tested: chloroquine 75 µM; HA2Pen fusion peptide 5 µM. |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | HeLa pLuc 705 (luciferase splice-correction reporter). Uptake also measured in CHO and N2a (SI). |
| Animal Model | |
| Administration Route | |
| Output Type | splice correction (luciferase reporter expression) |
| Output Value | |
| Output Units | |
| Output Notes | Negative control: no significant splice correction at 10 µM despite uptake (control for sequence specificity). |
| Toxicity Notes | LDH leakage: no substantial membrane disruption at tested doses. WST-1: no toxicity in complete medium; slight viability decrease for Tat-PNA and TP-PNA at 10 µM in serum-free conditions. |
| Curation Notes |