| Name | Ultevursen |
|---|
| 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 |
| Molecular Formula | C273H391N81O144P20S20 |
|---|---|
| Molecular Weight | 8372.24 |