TH.O.17 - Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes
| Autor: | Colella, P., Puzzo, F., Biferi, M., Bali, D., Paulk, N., Vidal, P., Collaud, F., Simon-Sola, M., Charles, S., Hardet, R., Leborgne, C., Sellier, P., van Wittenberghe, L., Boisgerault, F., Barkats, M., Laforêt, P., Kay, M., Koeberl, D., Ronzitti, G., Mingozzi, F. |
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| Zdroj: | In Neuromuscular Disorders October 2017 27 Supplement 2:S246-S246 |
| Databáze: | ScienceDirect |
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Full Text from ScienceDirect