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
Titel:
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
Auteur:
Ornaghi, Francesca Sala, Davide Tedeschi, Fabiana Maffia, Maria Chiara Bazzucchi, Martina Morena, Francesco Valsecchi, Manuela Aureli, Massimo Martino, Sabata Gritti, Angela