Development of non-viral vectors for neuronal-targeted delivery of CRISPR-Cas9 RNA-proteins (2022)
Sequence: CDIFTNSRGKRASNK-[Stearyl-AGYLLGKLLOOLAAAALOOLL-NH2]
| Experiment Id | EXP002444 |
|---|---|
| Paper | Development of non-viral vectors for neuronal-targeted delivery of CRISPR-Cas9 RNA-proteins |
| Peptide | C2-PepFect14 |
| Delivery Success Class | no |
| In Vivo Flag | no |
| Uptake Confirmed | yes |
| Label Confidence | high |
| In Vitro Functional Effect | yes |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | |
| Mixing Ratio | N/P ~3 |
| Formulation Format | polyplex |
| Formulation Components | C2-PF14 + Cas9 RNP |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vitro |
| Model Type | in vitro |
| Cell Lines Or Primary Cells | SH-SY5Y, M17, PC12 neuronal cells |
| Animal Model | |
| Administration Route | |
| Output Type | genome editing / protein knockdown |
| Output Value | Efficient SNCA editing and reduced alpha-synuclein expression |
| Output Units | |
| Output Notes | Functional CRISPR delivery with neuronal selectivity |
| Toxicity Notes | Low cytotoxicity in neuronal cells |
| Curation Notes |