Braxlosiran
Braxlosiran is a component of the AOC reagent Delpacibart braxlosiran (HY-177566), consisting of an siRNA targeting DUX4 (double homeobox protein) and SMCC linker (HY-42360). Delpacibart braxlosiran binds to TfR1. Aberrant expression of the DUX4 protein causes changes in gene expression in muscle cells, which are associated with muscle function loss in facioscapulohumeral muscular dystrophy (FSHD).
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- Molecular Weight:13884.20
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Speicherung:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
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Biologische Aktivität
Beschreibung
IC50 & Target
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Oligonucleotide-Linker Conjugates for AOC |
In Vitro
Delpacibart braxlosiran binds to TfR1[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
Chemical Information
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Appearance Solid
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Molecular Weight 13884.20
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Color White to off-white
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SMILES
[Braxlosiran]
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Versand
Room temperature in continental US; may vary elsewhere.
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Speicherung
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Reinheit & Dokumentation
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Data Sheet (274 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Handling Instructions (2659 KB)
Verweise
Calculators
Konzentration (Stammlösung) × Volumen (Stammlösung) = Konzentration (Ziellösung) × Volumen (Ziellösung)
Keywords
- Braxlosiran
- Antibody-Oligonucleotide Conjugates (AOCs)
- Small Interfering RNA (siRNA)
- DUX4-regulated gene expression
- cynomolgus monkey
- transferrin receptor 1
- DUX4 mRNA
- skeletal muscle cells
- HEK293 cells
- facioscapulohumeral muscular dystrophy
- FSHD patient-derived myotubes
- murine
- non-human primate
- Inhibitor
- inhibitor
- inhibit