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Precision rewriting of muscle genetics: therapeutic horizons of base and prime editing in skeletal muscle disorders.

Gene therapy - 1 May 2026

Saydam Selin, Dinçer Pervin

Abstract excerpt

Base Editing (BE) and Prime Editing (PE), novel precision tools of the CRISPR/Cas toolbox, have emerged as transformative technologies that enable highly specific genetic modifications. Their compatibility with post-mitotic cell types makes them invaluable for treating genetic skeletal muscle disorders. Despite their severity and progressive nature, monogenic muscle diseases remain without definitive treatments....

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CRISPR-Cas Systems
Gene Editing
Genetic Therapy
Muscle, Skeletal
Muscular Diseases

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 41345283
DOI: 10.1038/s41434-025-00574-1

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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