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A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease by P. Sellier, F. Collaud, Y. Krimi Benchekroun, V. Jimenez, X. Leon, N. Daniele, Q.H. Pham, J. El Andari, T. VandenDriessche, M.K. Chuah, D. Grimm, F. Bosch, F. Mingozzi, G. Ronzitti
Published 2025-06-01Get full text
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