Eteplirsen
Based on 1 Customer Validation
Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research.
Nos produits utilisent uniquement pour la recherche. Nous ne vendons pas aux patients.
- Pureté: 98.50%
- CAS No.: 1173755-55-9
- Formule: C364H569N177O122P30
- Masse moléculaire:10305.74
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Stockage:
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
To place orders, for customer services and technical support, please contact: MedChemExpress USA
Tel: 609-228-6898 E-mail: [email protected] [email protected]
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Activité biologique
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Chemical Information
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Solvant et solubilité
- Pureté et documentation
- Références
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Help & FAQs
Activité biologique
Eteplirsen acts to promote dystrophin production by restoring the translational reading frame of Duchenne muscular dystrophy (DMD) through specific skipping of exon 51 in defective gene variants[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
| NCT Number | Sponsor | Condition | Start Date |
Phase
|
|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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CAS No. 1173755-55-9
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Appearance Solid
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Masse moléculaire 10305.74
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Formule C364H569N177O122P30
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Color White to off-white
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SMILES
O=C1NC(N)=NC2=C1N=CN2[C@H]3CNC[C@H](O3)COP(N4C[C@H](O[C@@H](N5C(N=CN=C6N)=C6N=C5)C4)COP(N7C[C@@H](COP(N8C[C@@H](COP(N9C[C@@H](COP(N%10C[C@@H](COP(N%11C[C@@H](COP(N%12C[C@@H](COP(N%13C[C@@H](COP(N%14C[C@@H](COP(N%15C[C@@H](COP(N%16C[C@@H](COP(N%17C[C@@H](COP(N%18C[C@@H](COP(N%19C[C@@H](COP(N%20C[C@@H](COP(N%21C[C@@H](COP(N%22C[C@@H](COP(N%23C[C@@H](COP(N%24C[C@@H](COP(N%25C[C@@H](COP(N%26C[C@@H](COP(N%27C[C@@H](COP(N%28C[C@@H](COP(N%29C[C@@H](COP(N%30C[C@@H](COP(N%31C[C@@H](COP(N%32C[C@@H](COP(N%33C[C@@H](COP(N%34C[C@@H](COP(N%35CCN(C(OCCOCCOCCO)=O)CC%35)(N(C)C)=O)O[C@@H](N%36C(N=C(N)C=C%36)=O)C%34)(N(C)C)=O)O[C@@H](N%37C(NC(C(C)=C%37)=O)=O)C%33)(N(C)C)=O)O[C@@H](N%38C(N=C(N)C=C%38)=O)C%32)(N(C)C)=O)O[C@@H](N%39C(N=C(N)C=C%39)=O)C%31)(N(C)C)=O)O[C@@H](N%40C(N=CN=C%41N)=C%41N=C%40)C%30)(N(C)C)=O)O[C@@H](N%42C(N=CN=C%43N)=C%43N=C%42)C%29)(N(C)C)=O)O[C@@H](N%44C(N=C(N)C=C%44)=O)C%28)(N(C)C)=O)O[C@@H](N%45C(N=CN=C%46N)=C%46N=C%45)C%27)(N(C)C)=O)O[C@@H](N%47C(NC(C(C)=C%47)=O)=O)C%26)(N(C)C)=O)O[C@@H](N%48C(N=C(N)C=C%48)=O)C%25)(N(C)C)=O)O[C@@H](N%49C(N=CN=C%50N)=C%50N=C%49)C%24)(N(C)C)=O)O[C@@H](N%51C(N=CN=C%52N)=C%52N=C%51)C%23)(N(C)C)=O)O[C@@H](N%53C(N=C(N)NC%54=O)=C%54N=C%53)C%22)(N(C)C)=O)O[C@@H](N%55C(N=C(N)NC%56=O)=C%56N=C%55)C%21)(N(C)C)=O)O[C@@H](N%57C(N=CN=C%58N)=C%58N=C%57)C%20)(N(C)C)=O)O[C@@H](N%59C(N=CN=C%60N)=C%60N=C%59)C%19)(N(C)C)=O)O[C@@H](N%61C(N=C(N)NC%62=O)=C%62N=C%61)C%18)(N(C)C)=O)O[C@@H](N%63C(N=CN=C%64N)=C%64N=C%63)C%17)(N(C)C)=O)O[C@@H](N%65C(NC(C(C)=C%65)=O)=O)C%16)(N(C)C)=O)O[C@@H](N%66C(N=C(N)NC%67=O)=C%67N=C%66)C%15)(N(C)C)=O)O[C@@H](N%68C(N=C(N)NC%69=O)=C%69N=C%68)C%14)(N(C)C)=O)O[C@@H](N%70C(N=C(N)C=C%70)=O)C%13)(N(C)C)=O)O[C@@H](N%71C%72=C(N=C%71)C(N)=NC=N%72)C%12)(N(C)C)=O)O[C@@H](N%73C(NC(C(C)=C%73)=O)=O)C%11)(N(C)C)=O)O[C@@H](N%74C(NC(C(C)=C%74)=O)=O)C%10)(N(C)C)=O)O[C@@H](N%75C(NC(C(C)=C%75)=O)=O)C9)(N(C)C)=O)O[C@@H](N%76C(N=C(N)C=C%76)=O)C8)(N(C)C)=O)O[C@@H](N%77C(NC(C(C)=C%77)=O)=O)C7)(N(C)C)=O)(N(C)C)=O
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Synonyms
AVI 4658
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Sequence
RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate]
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Livraison
Room temperature in continental US; may vary elsewhere.
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Stockage
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
Solvant et solubilité
DMSO : 50 mg/mL (4.85 mM; Need ultrasonic; Hygroscopic DMSO has a significant impact on the solubility of product, please use newly opened DMSO)
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 (stored under nitrogen, 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.
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 (stored under nitrogen, 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.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Select the appropriate dissolution method based on your experimental animal and administration route.
- For the following dissolution methods, please ensure to first prepare a clear stock solution using an In Vitro approach and then sequentially add co-solvents:
- To ensure reliable experimental results, the clarified stock solution can be appropriately stored based on storage conditions. As for the working solution for In Vivo experiments, it is recommended to prepare freshly and use it on the same day.
- The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.
Add each solvent one by one: 10% DMSO 40% PEG300 5% Tween-80 45% Saline
Solubility: ≥ 1.25 mg/mL (0.12 mM); Clear solution
This protocol yields a clear solution of ≥ 1.25 mg/mL (saturation unknown).
Taking 1 mL working solution as an example, add 100 μL DMSO stock solution (12.5 mg/mL) to 400 μL PEG300, and mix evenly; then add 50 μL Tween-80 and mix evenly; then add 450 μL Saline to adjust the volume to 1 mL.
Preparation of Saline: Dissolve 0.9 g sodium chloride in ddH₂O and dilute to 100 mL to obtain a clear Saline solution.
Add each solvent one by one: 10% DMSO 90% (20% SBE-β-CD in Saline)
Solubility: ≥ 1.25 mg/mL (0.12 mM); Clear solution
This protocol yields a clear solution of ≥ 1.25 mg/mL (saturation unknown).
Taking 1 mL working solution as an example, add 100 μL DMSO stock solution (12.5 mg/mL) to 900 μL 20% SBE-β-CD in Saline, and mix evenly.
Preparation of 20% SBE-β-CD in Saline (4°C, storage for one week): 2 g SBE-β-CD powder is dissolved in 10 mL Saline, completely dissolve until clear.
For the following dissolution methods, please prepare the working solution directly:
It is recommended to prepare fresh solutions and use them promptly within a short period of time.
The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.
Add each solvent one by one: PBS
Solubility: 33.33 mg/mL (3.23 mM); Clear solution; Need ultrasonic
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.
Please enter your animal formula composition:
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%DMSO +
Recommended: Keep the proportion of DMSO in working solution below 2% if your animal is weak.
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%+
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+%Tween-80 + +
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%Saline +
The co-solvents required include: DMSO, . All of co-solvents are available by MedChemExpress (MCE). , Tween 80. All of co-solvents are available by MedChemExpress (MCE).
Working solution concentration: 0.22 mg/mL
Method for preparing stock solution: mg drug dissolved in μL DMSO. Stock solution concentration: mg/mL. * In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
1. Take μL DMSO stock solution;
2. Add μL .
μL , mix evenly;
3. Then add μL Tween 80, mix evenly;
4. Then add μL
Please ensure that the stock solution in the first step is dissolved to a clear state, and add co-solvents in sequence. You can use ultrasonic heating (ultrasonic cleaner, recommended frequency 20-40 kHz), vortexing, etc. to assist dissolution.
Pureté et documentation
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Fiche technique (270 KB)
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SDS (254 KB)
- English - EN (254 KB)
- Français - FR (254 KB)
- Deutsch - DE (254 KB)
- Norwegian - NO (254 KB)
- Español - ES (254 KB)
- Swedish - SV (254 KB)
- Italian - IT (254 KB)
- Korean - KR (254 KB)
- Portuguese - PT (254 KB)
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Instruction de manipulation (2242 KB)
Références
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 (stored under nitrogen, 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 |
|---|---|---|---|---|---|
| DMSO | 1 mM | 0.0970 mL | 0.4852 mL | 0.9703 mL | 2.4258 mL |
Eteplirsen Related Classifications
HY-108753 Related Classifications
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