Ultevursen (QR-421a) is a single-stranded RNA oligonucleotide that acts as a splice-switching agent, designed to promote the skipping of exon 13 in the USH2A gene, which encodes usherin. This mechanism aims to halt vision loss associated with retinitis pigmentosa caused by mutations within this exon. Ultevursen has potential applications in gene therapy and research focused on treatments for genetic retinal diseases.
Ultevursen (QR-421a) is a single-stranded RNA oligonucleotide that acts as a splice-switching agent, designed to promote the skipping of exon 13 in the USH2A gene, which encodes usherin. This mechanism aims to halt vision loss associated with retinitis pigmentosa caused by mutations within this exon. Ultevursen has potential applications in gene therapy and research focused on treatments for genetic retinal diseases.
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