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25. 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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28. Biochemical consequences of mutations causing the GM2 gangliosidoses. Mahuran DJ Biochim Biophys Acta; 1999 Oct; 1455(2-3):105-38. PubMed ID: 10571007 [TBL] [Abstract][Full Text] [Related]
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30. Adenoviral gene therapy of the Tay-Sachs disease in hexosaminidase A-deficient knock-out mice. Guidotti JE; Mignon A; Haase G; Caillaud C; McDonell N; Kahn A; Poenaru L Hum Mol Genet; 1999 May; 8(5):831-8. PubMed ID: 10196372 [TBL] [Abstract][Full Text] [Related]
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33. Mouse model of GM2 activator deficiency manifests cerebellar pathology and motor impairment. Liu Y; Hoffmann A; Grinberg A; Westphal H; McDonald MP; Miller KM; Crawley JN; Sandhoff K; Suzuki K; Proia RL Proc Natl Acad Sci U S A; 1997 Jul; 94(15):8138-43. PubMed ID: 9223328 [TBL] [Abstract][Full Text] [Related]
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