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Breaking News: 100 days of uncertaintySee today's BioWorld Science
Home » Targeting recurrent ABCA4 variant with antisense oligonucleotides as new strategy for Stargardt disease
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Ocular

Targeting recurrent ABCA4 variant with antisense oligonucleotides as new strategy for Stargardt disease

Feb. 3, 2025
Stargardt disease type 1 (STGD1) is an inherited retinal recessive disease caused by biallelic variants in the ABCA4 gene. One of the recurrent variants is located at the exon-intron junction of exon 6, c.768G>T. Due to its high prevalence, c.768G>T is an interesting therapeutic target for STGD1. Researchers from Radboud University developed a new antisense oligonucleotide (AON) therapy, designed to rescue the splicing defect caused by this variant.
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