RNA-based discovery and correction of splicing defects caused by POLR3A missense mutations

Molecular Therapy — Nucleic Acids 2026 Open access

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Authors: Lulzim Shkreta, Aurélie Delannoy, Johanne Toutant, James A. McSwiggen, Benoı̂t Chabot

DOI: 10.1016/j.omtn.2026.102850

Cited by: 1

References: 42

Abstract

missense mutations but also establish CRISPR-dCas13Rx as a useful tool for identifying and correcting disease-causing splicing aberrations.

RNA regulation and disease RNA Research and Splicing Nuclear Structure and Function

References 42