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1. Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes

Author

Colella, P., primary, Puzzo, F., additional, Biferi, M., additional, Bali, D., additional, Paulk, N., additional, Vidal, P., additional, Collaud, F., additional, Simon-Sola, M., additional, Charles, S., additional, Hardet, R., additional, Leborgne, C., additional, Sellier, P., additional, van Wittenberghe, L., additional, Boisgerault, F., additional, Barkats, M., additional, Laforêt, P., additional, Kay, M., additional, Koeberl, D., additional, Ronzitti, G., additional, and Mingozzi, F., additional

Published
2017
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