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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. Ornaghi F, et al. Among authors: gritti a. Neurobiol Dis. 2020 Feb;134:104667. doi: 10.1016/j.nbd.2019.104667. Epub 2019 Nov 1. Neurobiol Dis. 2020. PMID: 31682993 Free article.
Neural precursor cell cultures from GM2 gangliosidosis animal models recapitulate the biochemical and molecular hallmarks of the brain pathology.
Martino S, di Girolamo I, Cavazzin C, Tiribuzi R, Galli R, Rivaroli A, Valsecchi M, Sandhoff K, Sonnino S, Vescovi A, Gritti A, Orlacchio A. Martino S, et al. Among authors: gritti a. J Neurochem. 2009 Apr;109(1):135-47. doi: 10.1111/j.1471-4159.2009.05919.x. Epub 2009 Feb 20. J Neurochem. 2009. PMID: 19166507 Free article.
Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy.
Meneghini V, Lattanzi A, Tiradani L, Bravo G, Morena F, Sanvito F, Calabria A, Bringas J, Fisher-Perkins JM, Dufour JP, Baker KC, Doglioni C, Montini E, Bunnell BA, Bankiewicz K, Martino S, Naldini L, Gritti A. Meneghini V, et al. Among authors: gritti a. EMBO Mol Med. 2016 May 2;8(5):489-510. doi: 10.15252/emmm.201505850. Print 2016 May. EMBO Mol Med. 2016. PMID: 27025653 Free PMC article.
136 results