In a GLP study, Jofra Hernández and colleagues demonstrate that lentiviral vector-mediated HSPC gene therapy effectively corrects long-term X-linked chronic granulomatous disease in a mouse model of the disease. A small proportion of mice develops hematopoietic tumors originating from rare dominant clones harboring vector insertions near oncogenes.

Jofra Hernandez, R., Calabria, A., Sanvito, F., De Mattia, F., Farinelli, G., Scala, S., et al. (2021). Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy. MOLECULAR THERAPY, 29(1), 86-102 [10.1016/j.ymthe.2020.09.030].

Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy

Omrani M.;
2021

Abstract

In a GLP study, Jofra Hernández and colleagues demonstrate that lentiviral vector-mediated HSPC gene therapy effectively corrects long-term X-linked chronic granulomatous disease in a mouse model of the disease. A small proportion of mice develops hematopoietic tumors originating from rare dominant clones harboring vector insertions near oncogenes.
Articolo in rivista - Articolo scientifico
gene therapy; GLP; Good Laboratory Practice; inflammation; lentiviral vectors; mouse model; myelodysplastic syndrome; X-linked chronic granulomatosis disease;
English
2021
29
1
86
102
none
Jofra Hernandez, R., Calabria, A., Sanvito, F., De Mattia, F., Farinelli, G., Scala, S., et al. (2021). Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy. MOLECULAR THERAPY, 29(1), 86-102 [10.1016/j.ymthe.2020.09.030].
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10281/530490
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