Ulefnersen
Based on 1 publication(s) in Google Scholar
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) .
For research use only. We do not sell to patients.
- Purity: 99.22%
- CAS No.: 2589926-25-8
- Formula: C230H321N68O126P19S13
- Molecular Weight:7059.72
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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)
Publications Citing Use of MedChemExpress (MCE) Ulefnersen
MoreAll DNA/RNA Synthesis Isoforms
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Biological Activity
Fused-in sarcoma (FUS)
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:Newborn (postnatal day 1 (P1)) P517/WT mice [1]
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Dosage:20 μg
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Administration:Intracerebroventricular injection, Once in total
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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.
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Animal Model:Newborn (P1) MN-P517L/Δ14 mice[1]
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Dosage:20 μg
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Administration:Intracerebroventricular injection, Once in total
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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.
| NCT Number | Sponsor | Condition | Start Date |
Phase
|
|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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CAS No. 2589926-25-8
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Appearance Solid
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Molecular Weight 7059.72
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Formula C230H321N68O126P19S13
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Color White to off-white
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SMILES
[Ulefnersen]
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Synonyms
ION-363
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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)
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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)
Publications (1)
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Journal Impact Factor
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Most Recent
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Signal Transduct Target Ther
Targeting fused in sarcoma (FUS): a novel antisense strategy for treating idiopathic pulmonary fibrosis. [Abstract]2026 Feb 26;11(1):70. PMID: 41741410
Purity & Documentation
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Data Sheet (273 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
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)