Article

Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to reduce <i>ABCA4</i> splice variants in Stargardt disease

2024-03-09

Abstract excerpt

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...

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Literature Corpus work: 9bb7c07c-9977-507c-a34b-23372f67e19d
DOI: 10.1101/2024.03.08.584155

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