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Article

Skeletal muscle-directed gene therapy: hijacking the fusogenic properties of muscle cells

12 Sept 2023

Abstract excerpt

A recent study published in Cell by the Millay lab reports on an elegant strategy to expand the repertoire of lentiviral (LV) vectors from being the main delivery tool for ex vivo gene therapy to an in vivo applicable viral vector that specifically transduces skeletal muscle. 1 This very significant advance may result in novel therapeutics for patients suffering from various skeletal muscle diseases.

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PMID: 37699901
DOI: 10.1038/s41392-023-01584-4

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