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  3. Ulefnersen sodium

Ulefnersen sodium  (Synonyms: ION-363 sodium)

Cat. No.: HY-147410A Purity: 94.55%
Handling Instructions Technical Support

Ulefnersen sodium (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen sodium can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen sodium can be used in the research of Amyotrophic Lateral Sclerosis (ALS) .

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

DNA, d([2′-O-(2-methoxyethyl)]rG-sp-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU-G-sp-T-sp-m5C-sp-A-sp-m5C-sp-m5C-sp-T-sp-T-sp-T-sp-m5C-sp-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rA-sp-[2′-O-(2-methoxyethyl)]m5rC-sp-[2′-O-(2-methoxyethyl)]m5rC), sodium salt

Ulefnersen sodium Chemical Structure

CAS No. : 2589926-27-0

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Based on 1 publication(s) in Google Scholar

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Description

Ulefnersen sodium (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen sodium can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen sodium can be used in the research of Amyotrophic Lateral Sclerosis (ALS) [1].

In Vivo

Ulefnersen sodium (ION363) (Intracerebroventricular injection, 20 μg, Once in total) effectively reduces postnatal levels of FUS protein in the brain and spinal cord of P517/WT mice and reverses the detergent insolubility of RNA-binding proteins (RBPs) associated with mutant FUS toxicity. [1].
Ulefnersen sodium (Intracerebroventricular injection, 20 μg, Once in total) efficiently silences Fus and reduces postnatal levels of FUS protein in the brain and spinal cord of P517L and Δ14 heterozygous mice, delaying motor neuron degeneration. [1].

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

Animal Model: Newborn (postnatal day 1 (P1)) P517/WT mice [1]
Dosage: 20 μg
Administration: Intracerebroventricular injection, Once in total
Result: Showed an overall decrease in both wild-type and mutant FUS protein to approximately 20–50% of the levels observed in matched controls at 1 month of age, as determined by western immunoblot analysis of brain and spinal cord.
Decreased the levels of TDP-43, hnRNP A1, hnRNP U and UPF1 in the detergent-insoluble fractions.
Showed strong nuclear signals in all cells, including MNs at 1 and 4 months. However, signals for both Ulefnersen and NTC ASO dropped to near background levels by 6 months, as determined by analysis of anti-ASO immunostaining.
Animal Model: Newborn (P1) MN-P517L/Δ14 mice[1]
Dosage: 20 μg
Administration: Intracerebroventricular injection, Once in total
Result: Observed no MN loss and prevented muscle denervation and microgliosis at 4 months. Observed an increase in muscle denervation and microgliosis at 6 months, indicating that the onset of disease pathology was delayed rather than completely prevented. However, observed no MN degeneration at 6 months.
Molecular Weight

7477.40

Formula

C230H302N68O126P19S13Na19

CAS No.
Appearance

Solid

Color

White to off-white

SMILES

[Ulefnersen (sodium)]

Shipping

Room temperature in continental US; may vary elsewhere.

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
In Vitro: 

H2O : ≥ 25 mg/mL (3.34 mM)

*"≥" means soluble, but saturation unknown.

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 0.1337 mL 0.6687 mL 1.3374 mL
5 mM --- --- ---
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 (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.

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In Vivo Dissolution Calculator
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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 (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 0.1337 mL 0.6687 mL 1.3374 mL 3.3434 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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  • 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.

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Product Name:
Ulefnersen sodium
Cat. No.:
HY-147410A
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