Baliforsen
Based on 1 Customer Validation
Baliforsen (ISIS 5987690) is an antisense oligonucleotide (ASO) that inhibits DMPK mRNA. Baliforsen binds within exon 9 of the human DMPK transcript to promote RNase H1-mediated degradation Baliforsen can be used for the research of myotonic dystrophy type 1.
For research use only. We do not sell to patients.
- Purity: 98.12%
- CAS No.: 1698048-23-5
- Molecular Weight:5616.00
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Storage:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Biological Activity
Baliforsen binds complementarily to specific sequences (CUG repeats) on the toxic messenger RNA (mRNA) produced by the Type 1 Myotonic Dystrophy Protein Kinase (DMPK) gene. Subsequently, it recruits RNase H; RNase H is then activated and precisely cleaves the toxic mRNA strand bound to Baliforsen, thereby degrading it.
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
DNA, d(P-thio)([2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rC-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]m5C-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]m5C-G-A-A-T-G-T-m5C-m5C-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]G-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]A-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA)
Chemical Information
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CAS No. 1698048-23-5
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Appearance Solid
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Molecular Weight 5616.00
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Color White to off-white
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SMILES
[Baliforsen]
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Synonyms
ISIS 598769; IONIS 598769; BIIB 065; ISIS-DMPK-2.5Rx
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Solvent & Solubility
H2O : ≥ 100 mg/mL (17.81 mM)
* "≥" means soluble, but saturation unknown.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Please enter the basic information of animal experiments:
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Recommended: Prepare an additional quantity of animals to account for potential losses during experiments.
Working solution concentration: 0.22 mg/mL
This product has good water solubility, please refer to the measured solubility data in water/PBS/Saline for details.
Purity & Documentation
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Data Sheet (271 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)
- Portuguese - PT (252 KB)
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Handling Instructions (2242 KB)
References
Complete Stock Solution Preparation Table
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
| Optional Solvent | Concentration Solvent Mass | 1 mg | 5 mg | 10 mg | 25 mg |
|---|---|---|---|---|---|
| H2O | 1 mM | 0.1781 mL | 0.8903 mL | 1.7806 mL | 4.4516 mL |
| 5 mM | 0.0356 mL | 0.1781 mL | 0.3561 mL | 0.8903 mL | |
| 10 mM | 0.0178 mL | 0.0890 mL | 0.1781 mL | 0.4452 mL | |
| 15 mM | 0.0119 mL | 0.0594 mL | 0.1187 mL | 0.2968 mL |
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
- Baliforsen
- 1698048-23-5
- ISIS 598769
- IONIS 598769
- BIIB 065
- ISIS-DMPK-2.5Rx
- ISIS598769
- ISIS-598769
- IONIS598769
- IONIS 598769
- IONIS-598769
- BIIB065
- BIIB 065
- BIIB-065
- Ser/Thr Kinase
- muscle splicing events
- injection-site reactions
- DMPK mRNA
- RNase H1
- exon 9
- human DMPK transcript
- skeletal muscle
- smyotonic dystrophy type 1
- Inhibitor
- inhibitor
- inhibit