Ultevursen

Modify Date: 2024-09-21 17:35:23

Ultevursen Structure
Ultevursen structure
Common Name Ultevursen
CAS Number 2642382-41-8 Molecular Weight 8372.24
Density N/A Boiling Point N/A
Molecular Formula C273H391N81O144P20S20 Melting Point N/A
MSDS N/A Flash Point N/A

 Use of Ultevursen


Ultevursen (QR-421a) is a single-stranded RNA based oligonucleotide that is designed to skip exon 13 in the RNA with the aim to stop vision loss in people that have retinitis pigmentosa due to a mutation in exon 13 of the USH2A gene (encoding usherin). Ultevursen sequence: (P-thio)[2′-O-(2-methoxyethyl)](A-G-m5C-m5U-m5U-m5C-G-G-A-G-A-A-A-m5U-m5U-m5U-A-A-A-m5U-m5C) (ACI)[1].

 Names

Name Ultevursen

 Ultevursen Biological Activity

Description Ultevursen (QR-421a) is a single-stranded RNA based oligonucleotide that is designed to skip exon 13 in the RNA with the aim to stop vision loss in people that have retinitis pigmentosa due to a mutation in exon 13 of the USH2A gene (encoding usherin). Ultevursen sequence: (P-thio)[2′-O-(2-methoxyethyl)](A-G-m5C-m5U-m5U-m5C-G-G-A-G-A-A-A-m5U-m5U-m5U-A-A-A-m5U-m5C) (ACI)[1].
Related Catalog
Target

Usherin[1]

References

[1]. International Nonproprietary Names for Pharmaceutical Substances (INN). WHO Drug Information, Vol. 36, No. 2, 2022.

 Chemical & Physical Properties

Molecular Formula C273H391N81O144P20S20
Molecular Weight 8372.24
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