Efficacy of adeno-associated virus gene therapy in a MNGIE murine model enhanced by chronic exposure to nucleosides

Background: Preclinical studies have shown that gene therapy is a feasible approach to treat mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). However, the genetic murine model of the disease (Tymp/Upp1 double knockout, dKO) has a limited functional phenotype beyond the metabolic imbala...

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Autori principali:	Ferran Vila-Julià, Raquel Cabrera-Pérez, Yolanda Cámara, Miguel Molina-Berenguer, Silvia Lope-Piedrafita, Michio Hirano, Federico Mingozzi, Javier Torres-Torronteras, Ramon Martí
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2020-12-01
Serie:	EBioMedicine
Soggetti:	MNGIE Gene therapy Nucleosides Mitochondrial disease Thymidine phosphorylase
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2352396420305090
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