Article

First Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy

2023-11-24

Abstract excerpt

We report the first correction by Prime editing of a mutation in the RYR1 gene, paving the way to gene therapies for RYR1-related myopathies. The RYR1 gene codes for a calcium channel named Ryanodine receptor 1, which is expressed in skeletal muscle fibers. The failure of this channel causes muscle weakness in patients, which degenerates into motor disabilities. Currently, there are no effective treatments for the...

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Literature Corpus work: 3e4ca5b7-968f-5325-b41b-b2f7c0e2e22c
DOI: 10.20944/preprints202311.1585.v1

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