EXP002000

Paper

Targeted Activation of Cystic Fibrosis Transmembrane Conductance Regulator (2019)

Peptide

Sequence: GRKKRRQRRR

RNA

Gapmer antisense oligonucleotide (ASO; 20-mer)

All experiment fields

Experiment Id	EXP002000
Paper	Targeted Activation of Cystic Fibrosis Transmembrane Conductance Regulator
Peptide	HIV-1 Tat CPP (recombinant Tat peptide)
Delivery Success Class	no
In Vivo Flag	no
Uptake Confirmed	no
Label Confidence	high
In Vitro Functional Effect	yes
Endosomal Escape Evidence
Peptide Concentration	Tat CPP final concentration 200 mM (Tat:gapmer 200:10 mM) for electrostatic particles; incubated 2 h at 37°C before adding to cells; cells incubated 5 days before qPCR/ELISA readouts.
Rna Concentration	Gapmer final concentration 10 mM in Tat particle formulation (G10).
Mixing Ratio	Electrostatic particle: Tat CPP : gapmer final concentrations 200:10 mM (i.e., 20:1 molar ratio Tat:gapmer) after 2 h incubation at 37°C.
Formulation Format	Electrostatic CPP/ASO particles (Tat–gapmer complexes)
Formulation Components	Tat CPP (GRKKRRQRRR) complexed with BGas gapmer G10 (20-mer) via electrostatic binding.
Size Nm
Zeta Mv
Model Scope	in_vitro
Model Type	in vitro
Cell Lines Or Primary Cells	CFPAC (cystic fibrosis pancreatic adenocarcinoma) cells (F508del/F508del)
Animal Model
Administration Route
Output Type	In vitro functional RNA effect: BGas knockdown and CFTR activation (qRT-PCR); CFTR protein by ELISA; functional iodide transport assay (EYFP quench) with/without VX809+VX770
Output Value	CFTR mRNA increased after G10-Tat treatment (qRT-PCR; referenced in supplemental).
Output Units
Output Notes	Tat-formulated gapmer reported functional in CFPAC cells (supplementary figure referenced in main text).
Toxicity Notes	IL-6 induction: no differential increase vs scramble; LDH cytotoxicity: modest increase vs control; overall described as relatively non-immunogenic/toxic under tested conditions.
Curation Notes