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) [1].

Product Specifications

CAS Number

[2589926-25-8]

Product Name Alternative

ION-363

UNSPSC

12352208

Target

DNA/RNA Synthesis

Type

Oligonucleotides

Related Pathways

Cell Cycle/DNA Damage

Applications

Neuroscience-Neurodegeneration

Field of Research

Neurological Disease

Assay Protocol

https://www.medchemexpress.com/ulefnersen.html

Purity

99.22

Solubility

10 mM in DMSO

Smiles

[Ulefnersen]

Molecular Formula

C230H321N68O126P19S13

Molecular Weight

7059.72

References & Citations

[1]Korobeynikov VA, et al. Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis. Nat Med. 2022;28 (1) :104-116.