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Article

Gene therapies for RyR1-related myopathies.

Current opinion in pharmacology - 1 Feb 2023

Marty Isabelle, Beaufils Mathilde, Fauré Julien, Rendu John

Abstract excerpt

Myopathies related to variations in the RYR1 gene are genetic diseases for which the therapeutic options are sparse, in part because of the very large size of the gene and protein, and of the distribution of variations all along the sequence. Taking advantage of the progress made in the gene therapy field, different approaches can be applied to the different genetic variations, either at the mRNA level or...

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PMID: 36529094
DOI: 10.1016/j.coph.2022.102330

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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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