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AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy.
Morabito G, Giannelli SG, Ordazzo G, Bido S, Castoldi V, Indrigo M, Cabassi T, Cattaneo S, Luoni M, Cancellieri C, Sessa A, Bacigaluppi M, Taverna S, Leocani L, Lanciego JL, Broccoli V. Morabito G, et al. Among authors: cabassi t. Mol Ther. 2017 Dec 6;25(12):2727-2742. doi: 10.1016/j.ymthe.2017.08.004. Epub 2017 Aug 10. Mol Ther. 2017. PMID: 28882452 Free PMC article.