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TH.O.17 - Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes.

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Bibliographic Details
Title: TH.O.17 - Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes.
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
Description
ISSN:09608966
DOI:10.1016/j.nmd.2017.06.543