Ultevursen

Names

[ Name ]:
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]:

Research Areas >> Others

Signaling Pathways >> Others >> Others

[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

Related Compounds

The content on this webpage is sourced from various professional data sources. If you have any questions or concerns regarding the content, please feel free to contact service1@chemsrc.com.