Article

Prime Editing Enables High-Efficiency Correction of the Ryr1 T4706M Mutation: A Promising Therapeutic Approach for RyR1-Related Myopathies

2025-09-13

Abstract excerpt

Prime editing has emerged as a powerful genome-editing tool for precise correction of pathogenic mutations, offering a promising therapeutic approach for genetic myopathies. Here, we evaluate the correction efficiency of the T4706M mutation in the Ryr1 gene, which is implicated in severe skeletal muscle dysfunction. Using an optimized epegRNA design and RNA electroporation, we achieved a remarkable 80% editing ef...

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Literature Corpus work: efc077f2-70b6-5b56-8ae3-e5afd1ae0982
DOI: 10.1101/2025.09.12.675683

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