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2227173-68-2

2227173-68-2 structure

2227173-68-2 structure

Name: Sepofarsen
Chemical Name: Sepofarsen
CAS Number: 2227173-68-2
Molecular Formula:
Molecular Weight:
Catalog: Research Areas Others
Create Date: 2023-08-26 18:07:31
Modify Date: 2025-08-26 15:51:26
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.

Name	Sepofarsen

Description	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.
Related Catalog	Research Areas >> Others Signaling Pathways >> Others >> Others
References	[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.

No Any Chemical & Physical Properties

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