2. Cell Cycle/DNA Damage
  3. Nucleoside Antimetabolite/Analog
  4. Viltolarsen

Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research.

The free form of the compound is prone to instability, it is advisable to consider the stable salt form (Viltolarsen sodium) that retains the same biological activity.

For research use only. We do not sell to patients.

RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-Gm5U-m5U-C)

Viltolarsen Chemical Structure

CAS No. : 2055732-84-6

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Viltolarsen Related Antibodies

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  • Purity & Documentation

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Description

Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen has the potential for Duchenne muscular dystrophy (DMD) research[1][2].

In Vitro

Viltolarsen (NS-065/NCNP-01; 2 days) induces exon 53 skipping in cells from a DMD model with a deletion of exons 45-52 with an EC50 value of 0.63 μM and in cells with a deletion of exons 48-52 with an EC50 value of 2.3 μM[2].
Viltolarsen (10 μM; 3 days) causes the dystrophin protein expression in cells from a DMD model[2].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

Viltolarsen Related Antibodies
Clinical Trial
Molecular Weight

6924.80

CAS No.
Appearance

Solid

Color

White to off-white

Sequence

RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-Gm5U-m5U-C)
SMILES

[Viltolarsen]
Shipping

Room temperature in continental US; may vary elsewhere.
Storage

-20°C, stored under nitrogen, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)

Solvent & Solubility
In Vitro: 

H2O : 100 mg/mL (14.44 mM; Need ultrasonic)

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 0.1444 mL 0.7220 mL 1.4441 mL
5 mM 0.0289 mL 0.1444 mL 0.2888 mL
View the 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.

* 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.

  • Molarity Calculator

  • Dilution Calculator

Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

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Volume
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Concentration (start) × Volume (start) = Concentration (final) × Volume (final)

This equation is commonly abbreviated as: C1V1 = C2V2

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C2

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In Vivo:

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.

  • Protocol 1

    Add each solvent one by one:  PBS

    Solubility: 50 mg/mL (7.22 mM); Clear solution; Need ultrasonic

In Vivo Dissolution Calculator
Please enter the basic information of animal experiments:

Dosage

mg/kg

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(per animal)

g

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(per animal)

μL

Number of animals

Recommended: Prepare an additional quantity of animals to account for potential losses during experiments.
Calculation results:
Working solution concentration: mg/mL
This product has good water solubility, please refer to the measured solubility data in water/PBS/Saline for details.
The concentration of the stock solution you require exceeds the measured solubility. The following solution is for reference only.If necessary, please contact MedChemExpress (MCE).
Purity & Documentation
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 (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
H2O 1 mM 0.1444 mL 0.7220 mL 1.4441 mL 3.6102 mL
5 mM 0.0289 mL 0.1444 mL 0.2888 mL 0.7220 mL
10 mM 0.0144 mL 0.0722 mL 0.1444 mL 0.3610 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.

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Viltolarsen Related Classifications

Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

Viltolarsen2055732-84-6NS-065/NCNP-01Nucleoside Antimetabolite/AnalogexonskippingDuchennemusculardystrophyphosphorodiamidatemorpholinoantisenseoligonucleotide53Inhibitorinhibitorinhibit

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