Enhancing the potency of in vivo lentiviral vector mediated gene therapy to hepatocytes

Enhancing the potency of in vivo lentiviral vector mediated gene therapy to hepatocytes

Abstract In vivo gene therapy to the liver using lentiviral vectors (LV) may represent a one-and-done therapeutic approach for monogenic diseases. Increasing LV gene therapy potency is crucial for reducing the effective doses, thus alleviating dose-dependent toxicities and facilitating manufacturing...

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Main Authors: Cesare Canepari, Michela Milani, Chiara Simoni, Francesco Starinieri, Monica Volpin, Anna Fabiano, Mauro Biffi, Fabio Russo, Rossana Norata, Martina Rocchi, Chiara Brombin, Federica Cugnata, Eugenio Montini, Francesca Sanvito, Markus Grompe, Alessio Cantore
Format: Article
Language:English
Published: Nature Portfolio 2025-05-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-60073-0
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