Sequence: AcRXRRBRRXRRBRXB-OH
| Experiment Id | EXP002354 |
|---|---|
| Paper | Systemic delivery of a peptide-linked morpholino oligonucleotide neutralizes mutant RNA toxicity in |
| Peptide | Peptide B |
| Delivery Success Class | yes |
| In Vivo Flag | yes |
| Uptake Confirmed | no |
| Label Confidence | high |
| In Vitro Functional Effect | |
| Endosomal Escape Evidence | |
| Peptide Concentration | |
| Rna Concentration | |
| Mixing Ratio | |
| Formulation Format | peptide–morpholino conjugate (PPMO) |
| Formulation Components | Peptide B-CAG25 PPMO |
| Size Nm | |
| Zeta Mv | |
| Model Scope | in_vivo |
| Model Type | in vivo |
| Cell Lines Or Primary Cells | |
| Animal Model | HSALR transgenic mouse model of myotonic dystrophy type 1 |
| Administration Route | intravenous (tail vein), weekly ×6 |
| Output Type | phenotypic rescue and molecular correction |
| Output Value | Correction of RNA splicing (Serca-1, ClC-1, Titin, Zasp); elimination of myotonia |
| Output Units | |
| Output Notes | Redistribution of Mbnl1 from nuclear foci; normalization of EMG signals |
| Toxicity Notes | No overt toxicity; normal ALT/AST, BUN, creatinine |
| Curation Notes |