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Pathways Recommended:	Stem Cell/Wnt Cell Cycle/DNA Damage

Results for "

WERI-Rb1 cells

" in MedChemExpress (MCE) Product Catalog:

By Targets:	DNA/RNA Synthesis (1) Nucleoside Antimetabolite/Analog (1)
By Research Areas:	Neurological Disease (1)
Oligonucleotides:	Antisense Oligonucleotides (1)

Targets Recommended: Nuclear Factor of activated T Cells (NFAT) TREM receptor

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

Ultevursen QR-421a	Nucleoside Antimetabolite/Analog DNA/RNA Synthesis	Neurological Disease
Ultevursen (QR-421a) is a splice-modulating antisense oligonucleotide targeting exon 13 of the USH2A gene, which restores the functional expression of Usherin protein by inducing exon skipping. Ultevursen binds to USH2A pre-mRNA and modulates the splicing process to specifically skip exon 13 carrying the pathogenic mutation c.2299delG, generating an in-frame transcript and a truncated yet functionally normal protein. Ultevursen exhibits concentration-dependent exon skipping activity in human cells and retinal organoid models, and restores Usherin expression and retinal function in zebrafish and gene-edited mouse models. Ultevursen can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .

Cat. No.	Product Name		Classification

Ultevursen QR-421a		Antisense Oligonucleotides
Ultevursen (QR-421a) is a splice-modulating antisense oligonucleotide targeting exon 13 of the USH2A gene, which restores the functional expression of Usherin protein by inducing exon skipping. Ultevursen binds to USH2A pre-mRNA and modulates the splicing process to specifically skip exon 13 carrying the pathogenic mutation c.2299delG, generating an in-frame transcript and a truncated yet functionally normal protein. Ultevursen exhibits concentration-dependent exon skipping activity in human cells and retinal organoid models, and restores Usherin expression and retinal function in zebrafish and gene-edited mouse models. Ultevursen can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa .

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