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 No.: 2409088-11-3
- Formule: C280H440N119O105P23
- Masse moléculaire:7865.60
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Stockage:
Please store the product under the recommended conditions in the Certificate of Analysis.
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Activité biologique
Description
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 No. 2409088-11-3
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Masse moléculaire 7865.60
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Formule 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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Livraison
Room temperature in continental US; may vary elsewhere.
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Stockage
Please store the product under the recommended conditions in the Certificate of Analysis.
Pureté et documentation
Références
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)