2. Oligonucleotides
  3. Antisense Oligonucleotides
  4. Ulefnersen

Ulefnersen (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 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 can be used in the research of Amyotrophic Lateral Sclerosis (ALS) .

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

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)

Ulefnersen Chemical Structure

CAS No. : 2589926-25-8

Size Price Stock
1 mg USD 143 Ask For Quote & Lead Time
5 mg USD 391 Ask For Quote & Lead Time
10 mg USD 607 Ask For Quote & Lead Time

* Please select Quantity before adding items.

This product is a controlled substance and not for sale in your territory.

Other Forms of Ulefnersen:

Ulefnersen Related Antibodies

Top Publications Citing Use of Products

  • Biological Activity

  • Purity & Documentation

  • References

  • Customer Review

Description

Ulefnersen (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 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 can be used in the research of Amyotrophic Lateral Sclerosis (ALS) [1].

IC50 & Target

Fused-in sarcoma (FUS)
In Vivo

Ulefnersen (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 (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

7059.72

Formula

C230H321N68O126P19S13
CAS No.
Appearance

Solid

Color

White to off-white

Sequence

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)
SMILES

[Ulefnersen]
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)

Purity & Documentation
References
  • No file chosen (Maximum size is: 1024 Kb)
  • If you have published this work, please enter the PubMed ID.
  • Your name will appear on the site.

Ulefnersen Related Classifications

  • Molarity Calculator

  • Dilution Calculator

The molarity calculator equation

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

Mass   Concentration   Volume   Molecular Weight *
= × ×

The dilution calculator equation

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

This equation is commonly abbreviated as: C1V1 = C2V2

Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
× = ×
C1   V1   C2   V2
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:

Ulefnersen2589926-25-8ION-363ION363ION 363OthersAntisense OligonucleotideASOFused-in sarcomaFUSAmyotrophic Lateral SclerosisALSNeurological diseaseInhibitorinhibitorinhibit

Your Recently Viewed Products:

Inquiry Online

Your information is safe with us. * Required Fields.

Product Name

  

Salutation

Applicant Name *

 

Email Address *

Phone Number *

 

Organization Name *

Department *

 

Requested quantity *

Country or Region *

      

Remarks

Bulk Inquiry

Inquiry Information

Product Name:
Ulefnersen
Cat. No.:
HY-147410
Quantity:
MCE Japan Authorized Agent:

Customer Review

Thank You for Your Feedback!

Request for HNMR Report

Please fill out this form to request the QC report. We will send it to your Email address shortly.

Your information is safe with us. * Required Fields.

Product Name

  

Lot #

Applicant Name *

 

Email Address *

Phone Number

 

Department *

Organization Name *

 

Country or Region *

Remarks

Request for HNMR Report

We have received your request and will respond to you as soon as possible.