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EXP001804

Paper

Peptide/Cas9 nanostructures for ribonucleoprotein cell membrane transport and gene edition (2017)

Peptide

PT24 (oleic aldehyde hydrazone amphiphile from peptide P)

Sequence: Arg/Leu-rich cationic peptide scaffold with two hydrazide (Hy) residues (exact sequence not in main text; schematic shows RRLKRRLLRRLKRL)

RNA

gRNA (CRISPR guide RNA) in Cas9 RNP

All experiment fields

Experiment Id EXP001804
Paper Peptide/Cas9 nanostructures for ribonucleoprotein cell membrane transport and gene edition
Peptide PT24 (oleic aldehyde hydrazone amphiphile from peptide P)
Delivery Success Class no
In Vivo Flag no
Uptake Confirmed yes
Label Confidence medium
In Vitro Functional Effect yes
Endosomal Escape Evidence yes
Peptide Concentration PT24 10 mM (optimized); also tested 0.5–12.5 µM for gel retardation; uptake studies at 5 µM TmPT24
Rna Concentration Cas9 RNP 40–160 nM (dose-response); typical 80 nM or 160 nM; peptide:RNP ratio 62.5:1 (mol/mol) used in T7E1 assays
Mixing Ratio RNP:PT24 = 1:62.5 (mol/mol) (editing assays)
Formulation Format Non-covalent peptide amphiphile/Cas9 ribonucleoprotein nanoparticles
Formulation Components PT24 amphiphilic CPP + SpCas9 protein complexed with gRNA (RNP); TAMRA-labelled PT24 (TmPT24) for uptake; DexPT24 for GR translocation assay
Size Nm 270.00
Zeta Mv 4.20
Model Scope in_vitro
Model Type in vitro
Cell Lines Or Primary Cells HeLa-EGFP
Animal Model
Administration Route
Output Type Genome editing (EGFP knockout; % EGFP-negative cells by flow cytometry, 3 days)
Output Value Screening shows EGFP-negative cells depending on aldehyde tail; PT24 (oleic aldehyde) best hit (typically >30% EGFP-negative in screen)
Output Units
Output Notes Hydrazone tail screening (PTn) identified T24 (oleic aldehyde) as best for EGFP editing; single incubation protocol.
Toxicity Notes Lower toxicity vs Lipofectamine 2000 at comparable editing (MTT).
Curation Notes