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Genetic mutations in HSV-1 replication-defective vectors: Implications for their safety in gene therapy applications.

Gene therapy - 1 Dec 2025

Cattaneo Stefano, Bettegazzi Barbara, Ingusci Selene, Verlengia Gianluca, Tascini Anna Sofia, Zucchini Silvia, Codazzi Franca, Morelli Marco J, Marzulli Marco, Glorioso Joseph C, Simonato Michele

Abstract excerpt

Beyond its well-known role in orofacial recurrent infections, HSV-1 has garnered significant attention in neuroscience for contrasting reasons. On one hand, it has been found to be involved in neurodegenerative processes; on the other, it may represent a versatile platform for gene therapy of brain diseases, due to its large genome that enables the delivery of sizable or multiple genes. These opposite features...

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Identifiers

PMID: 41016924
DOI: 10.1038/s41434-025-00566-1
PMCID: PMC12714583

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