Antisense Oligonucleotide-Based Rescue of Aberrant Splicing Defects Caused by 15 Pathogenic Variants in <i>ABCA4</i>

Antisense Oligonucleotide-Based Rescue of Aberrant Splicing Defects Caused by 15 Pathogenic Variants in <i>ABCA4</i>

The discovery of novel intronic variants in the <i>ABCA4</i> locus has contributed significantly to solving the missing heritability in Stargardt disease (STGD1). The increasing number of variants affecting pre-mRNA splicing makes <i>ABCA4</i> a suitable candidate for antisen...

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Bibliographic Details
Main Authors: Tomasz Z. Tomkiewicz, Nuria Suárez-Herrera, Frans P. M. Cremers, Rob W. J. Collin, Alejandro Garanto
Format: Article
Language:English
Published: MDPI AG 2021-04-01
Series:International Journal of Molecular Sciences
Subjects:
antisense oligonucleotide
<i>ABCA4</i>
Stargardt disease
inherited retinal diseases
splicing modulation
RNA therapy
Online Access:https://www.mdpi.com/1422-0067/22/9/4621

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