Brogidirsen
Brogidirsen (NS 089; NCNP 02) is a dual-targeting antisense oligonucleotide capable of inducing AAexon skipping. Brogidirsen consists of two 12-mer phosphorodiamidate morpholino oligomers (PMOs) linked directly together. Through these PMOs, Brogidirsen targets regenerating muscle fibers, facilitating the reprogramming of cells that overexpress MYOD1 into the myogenic lineage. Brogidirsen is used in research related to Duchenne muscular dystrophy.
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- CAS. Nr.: 2409088-11-3
- Formel: C280H440N119O105P23
- Molecular Weight:7865.60
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Speicherung:
Please store the product under the recommended conditions in the Certificate of Analysis.
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Biologische Aktivität
Beschreibung
IC50 & Target
Target: Dystrophin Synthesis[1]
In Vitro
Brogidirsen (1-10 μM; 72 h) induces dose-dependent exon 44 skipping and dystrophin protein expression in donor-derived MYOD1-UDCs, myotubes, and MYOD1-transduced fibroblasts from DMD patients amenable to exon 44 skipping[2].
Brogidirsen (1-10 μM) exhibits an EC50 of 7.12 μM for exon 44 skipping in healthy donor-derived MYOD1-UDCs[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Cell Line:healthy donor-derived MYOD1-UDCs
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Concentration:10 μM
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Incubation Time:/
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Result:Increased exon 44 skipping expression.
Chemical Information
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CAS. Nr. 2409088-11-3
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Molecular Weight 7865.60
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Formel C280H440N119O105P23
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SMILES
[Brogidirsen]
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Synonyms
NS 089; NCNP 02
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Sequence
RNA, [P-deoxy-P-(dimethylamino)](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco)(2′a→5′)(m5U-m5U-G-m5U-G-m5U-C-m5U-m5U-m5U-C-m5U-m5U-C-m5U-G-m5U-m5U-A-G-C-C-A-C)
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Versand
Room temperature in continental US; may vary elsewhere.
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Speicherung
Please store the product under the recommended conditions in the Certificate of Analysis.
Reinheit & Dokumentation
Verweise
Calculators
Konzentration (Stammlösung) × Volumen (Stammlösung) = Konzentration (Ziellösung) × Volumen (Ziellösung)