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Article

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

Cells - 22 Dec 2023

Godbout Kelly, Rousseau Joël, Tremblay Jacques P

Abstract excerpt

We report the first correction from prime editing 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 leads to motor disabilities. Currently, there are no effective treatments for these...

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Identifiers

PMID: 38201236
DOI: 10.3390/cells13010031
PMCID: PMC10777931

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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