TH.O.17 - Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes.
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| Title: | TH.O.17 - Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes. |
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| Authors: | Colella, P.1, Puzzo, F.1, Biferi, M.2, Bali, D.3, Paulk, N.4, Vidal, P.1, Collaud, F.1, Simon-Sola, M.1, Charles, S.1, Hardet, R.2, Leborgne, C.1, Sellier, P.1, Van Wittenberghe, L.1, Boisgerault, F.1, Barkats, M.2, Laforêt, P.5, Kay, M.4, Koeberl, D.3, Ronzitti, G.1, Mingozzi, F.6 |
| Source: | Neuromuscular Disorders. Oct2017 Supplement 2, Vol. 27, pS246-S246. 1p. |
| Database: | Academic Search Ultimate |
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