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Sepofarsen

Sepofarsen (QR-110) is an RNA antisense oligonucleotide targeting to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene.

Product Specifications

CAS Number

2227173-68-2

Product Name Alternative

QR 110

UNSPSC

12352208

Target

Others

Type

Oligonucleotides

Related Pathways

Others

Field of Research

Others

Assay Protocol

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

Solubility

10 mM in H2O

Smiles

[Sepofarsen]

Molecular Formula

C180H235N67O101P16S

Molecular Weight

5962.00

References & Citations

[1]Russell SR, Drack AV, Cideciyan AV, et al. Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial. Nat Med. 2022;28 (5) :1014-1021.|[2]Cideciyan AV, Jacobson SG, Ho AC, et al. Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report. Nat Med. 2021;27 (5) :785-789.

Shipping Conditions

Room temperature

Scientific Category

Oligonucleotides

Clinical Information

Phase 3

Frequently Asked Questions

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