A novel hybrid promote directing AAV-mediated expression of acid alpha-glucosidase to liver, muscle and CNS yields optimized outcomes in a mouse model of Pompe disease

G. Heffner,L. James, C. Chaivorapol, M. Mei, T. Stinchcombe,J. Ton,B. Schoser,H. Meng,M. Lawlor,S. Prasad,J. Gray

MOLECULAR THERAPY(2018)

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