2. Cell Cycle/DNA Damage Others
  3. DNA/RNA Synthesis Others
  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

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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
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Keywords:

Ulefnersen2589926-25-8ION-363ION363ION 363DNA/RNA SynthesisOthersAntisense OligonucleotideASOFused-in sarcomaFUSAmyotrophic Lateral SclerosisALSNeurological diseaseInhibitorinhibitorinhibit

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