Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm ) ameliorates disease in a murine model of McArdle disease

Elyshia L McNamara, Rhonda L Taylor, Joshua S Clayton, Hayley Goullee, Kimberley L Dilworth, Tomàs Pinós, Astrid Brull, Ian E Alexander, Leszek Lisowski, Gianina Ravenscroft, Nigel G Laing, Kristen J Nowak

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Medicine & Life Sciences