2024
DOI: 10.1101/2024.03.08.584155
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Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to reduceABCA4splice variants in Stargardt disease

Roxanne Hsiang-Chi Liou,
Daniel Urrutia-Cabrera,
Ida Maria Westin
et al.

Abstract: Dysregulation of the alternative splicing process results in aberrant mRNA transcripts, leading to dysfunctional proteins or nonsense-mediated decay that cause a wide range of mis-splicing diseases. Development of therapeutic strategies to target the alternative splicing process could potentially shift the mRNA splicing from disease isoforms to a normal isoform and restore functional protein. As a proof of concept, we focus on Stargardt disease (STGD1), an autosomal recessive inherited retinal disease caused b… Show more

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