Intercellular transfer of the virally derived precursor form of acid α-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy pompe disease

D. F. Pauly, T. J. Fraites, C. Toma, H. S. Bayes, M. L. Huie, R. Hirschhorn, P. H. Plotz, N. Raben, P. D. Kessler, B. J. Byrne

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Medicine & Life Sciences