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10.1038/mtm.2015.38 http://scihub22266oqcxt.onion/10.1038/mtm.2015.38 C4605225!4605225 !26509184     free     free     free Warning: file_get_contents(https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&id=26509184 &cmd=llinks): Failed to open stream: HTTP request failed! 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Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy |pdf=|usr=}}{{26509184 }} gab.com Text Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy JO: Mol Ther Methods Clin Dev http://www.kidney.de/pget.php?&tw=1&pmid=26509184 #FOAvip Globoid cell leukodystrophy (GLD) is a demyelinating lysosomal storage disease due to the deficiency of the galactocerebrosidase (GALC) enzyme. The favorable outcome of hematopoietic stem and progenitor cell (HSPC)-based approaches in GLD and other similar diseases suggests HSPC gene therapy as a promising therapeutic option for patients. The path to clinical development of this strategy was hampered by a selective toxicity of the overexpressed GALC in the HSPC compartment. Here, we presented the optimization of a lentiviral vector (LV) in which miR-126 regulation was coupled to codon optimization of the human GALC cDNA to obtain a selective and enhanced enzymatic activity only upon transduced HSPCs differentiation. The safety of human GALC overexpression driven by this LV was extensively demonstrated in vitro and in vivo on human HSPCs from healthy donors. No perturbation in the content of proapoptotic sphingolipids, gene expression profile, and capability of engraftment and mutlilineage differentiation in chimeric mice was observed. The therapeutic potential of this LV was then assessed in a severe GLD murine model that benefited from transplantation of corrected HSPCs with longer survival and ameliorated phenotype as compared to untreated siblings. This construct has thus been selected as a candidate for clinical translation. Twit Text FOAVip Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy ????Mol Ther Methods Clin Dev http://www.kidney.de/pget.php?&tw=1&pmid=26509184 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=26509184 #FOAvip English Wikipedia <ref>{{Cite pmid|26509184 }}</ref> Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy #MMPMID26509184 Ungari S ; Montepeloso A ; Morena F ; Cocchiarella F ; Recchia A ; Martino S ; Gentner B ; Naldini L ; Biffi A Mol Ther Methods Clin Dev 2015[]; 2 (ä): 15038 PMID26509184 show ga Globoid cell leukodystrophy (GLD) is a demyelinating lysosomal storage disease due to the deficiency of the galactocerebrosidase (GALC) enzyme. The favorable outcome of hematopoietic stem and progenitor cell (HSPC)-based approaches in GLD and other similar diseases suggests HSPC gene therapy as a promising therapeutic option for patients. The path to clinical development of this strategy was hampered by a selective toxicity of the overexpressed GALC in the HSPC compartment. Here, we presented the optimization of a lentiviral vector (LV) in which miR-126 regulation was coupled to codon optimization of the human GALC cDNA to obtain a selective and enhanced enzymatic activity only upon transduced HSPCs differentiation. The safety of human GALC overexpression driven by this LV was extensively demonstrated in vitro and in vivo on human HSPCs from healthy donors. No perturbation in the content of proapoptotic sphingolipids, gene expression profile, and capability of engraftment and mutlilineage differentiation in chimeric mice was observed. The therapeutic potential of this LV was then assessed in a severe GLD murine model that benefited from transplantation of corrected HSPCs with longer survival and ameliorated phenotype as compared to untreated siblings. This construct has thus been selected as a candidate for clinical translation. äDesign of a regulated lentiviral vector for hematopoietic stem cell ge(epmc).pdf DeepDyve Pubget Overpricing