Article

Functional benefit of CRISPR/Cas9-induced allele deletion for <i>RYR1</i> dominant mutation

2024-01-25

Abstract excerpt

More than 700 pathogenic or probably pathogenic variations have been identified in the RYR1 gene causing various myopathies collectively known as “ RYR1 -related myopathies”. Currently, there is no treatment for these myopathies, and gene therapy stands out as one of the most promising approaches. In the context of a dominant form of Central Core Disease due to a RYR1 mutation, we aimed at showing the functiona...

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Literature Corpus work: c93b891c-a909-51ae-84c9-2ad8f01ee063
DOI: 10.1101/2024.01.24.576997

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