Correction of the Splicing Defect Caused by a Recurrent Variant in <i>ABCA4</i> (c.769-784C>T) That Underlies Stargardt Disease

Correction of the Splicing Defect Caused by a Recurrent Variant in <i>ABCA4</i> (c.769-784C>T) That Underlies Stargardt Disease

Stargardt disease is an inherited retinal disease caused by biallelic mutations in the <i>ABCA4</i> gene, many of which affect <i>ABCA4</i> splicing. In this study, nine antisense oligonucleotides (AONs) were designed to correct pseudoexon (PE) inclusion caused by a recurrent...

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Bibliographic Details
Main Authors: Tomasz Z. Tomkiewicz, Sara E. Nieuwenhuis, Frans P. M. Cremers, Alejandro Garanto, Rob W. J. Collin
Format: Article
Language:English
Published: MDPI AG 2022-12-01
Series:Cells
Subjects:
antisense oligonucleotides
<i>ABCA4</i>
Stargardt disease
inherited retinal disease
splicing modulation
RNA therapy
Online Access:https://www.mdpi.com/2073-4409/11/24/3947

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