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EXP001213

Paper

Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice (2008)

Peptide

CPP-B conjugate (mis-23D-B, mismatched PMO control)

Sequence: RXRRBRRXRRBRXB

RNA

PMO (morpholino antisense oligo)

All experiment fields

Experiment Id EXP001213
Paper Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of
Peptide CPP-B conjugate (mis-23D-B, mismatched PMO control)
Delivery Success Class no
In Vivo Flag yes
Uptake Confirmed no
Label Confidence high
In Vitro Functional Effect
Endosomal Escape Evidence
Peptide Concentration
Rna Concentration 12 mg/kg/day (PPMO dose) for 4 days
Mixing Ratio
Formulation Format covalent peptide–PMO conjugate (PPMO)
Formulation Components CPP-B conjugated to mismatched PMO (mis-23D-B)
Size Nm
Zeta Mv
Model Scope in_vivo
Model Type in vivo
Cell Lines Or Primary Cells
Animal Model mdx mouse (DMD model) / EGFP-654 (negative control for imaging)
Administration Route intravenous (IV)
Output Type negative control
Output Value No exon 23-skipped transcript / no reporter upregulation (control).
Output Units
Output Notes Mismatched control reported as negative (no exon 23–skipped transcript in mdx; no EGFP upregulation in imaging control).
Toxicity Notes
Curation Notes