NeuroscienceBrainPeer-reviewed
Gene replacement therapy provides benefit in an adult mouse model of Leigh syndrome
Robin Reynaud-Dulaurier, Giorgia Benegiamo, Elena Marrocco…·Mickaël Decressac
This paper demonstrates that gene replacement therapy using AAV-PHP.B vector improves survival and corrects metabolic and behavioral deficits in a mouse model of Leigh syndrome.
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