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26. Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis. Ornaghi F; Sala D; Tedeschi F; Maffia MC; Bazzucchi M; Morena F; Valsecchi M; Aureli M; Martino S; Gritti A Neurobiol Dis; 2020 Feb; 134():104667. PubMed ID: 31682993 [TBL] [Abstract][Full Text] [Related]
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