| Company Name: |
TargetMol Chemicals Inc.
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| Tel: |
15002134094 |
| Email: |
marketing@targetmol.cn |
| Products Intro: |
Product Name:Ulefnersen
CAS:2589926-25-8
Package:50mg;10mg
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| | Ulefnersen Basic information |
| | Ulefnersen Chemical Properties |
| form | 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) |
| | Ulefnersen Usage And Synthesis |
| Uses | 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]. | | 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]. | 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.
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| 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. |
| | References | [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. DOI:10.1038/s41591-021-01615-z |
| | Ulefnersen Preparation Products And Raw materials |
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