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Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: Implications for in vivo end ex vivo gene therapy of GM2 gangliosidosis
Ornaghi, Francesca, Sala, Davide, Tedeschi, Fabiana, Maffia, Maria Chiara, Bazzucchi, Martina, Morena, Francesco, Valsecchi, Manuela, Aureli, Massimo, Martino, Sabata, Gritti, AngelaLanguage:
english
Journal:
Neurobiology of Disease
DOI:
10.1016/j.nbd.2019.104667
Date:
November, 2019
File:
PDF, 15.00 MB
english, 2019