Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy
| Title: | Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy |
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| Authors: | Jofra Hernandez R.; Calabria A.; Sanvito F.; De Mattia F.; Farinelli G.; Scala S.; Visigalli I.; Carriglio N.; De Simone M.; Vezzoli M.; Cecere F.; Migliavacca M.; Basso-Ricci L.; Omrani M.; Benedicenti F.; Norata R.; Rancoita P. M. V.; Di Serio C.; Albertini P.; Cristofori P.; Naldini L.; Gentner B.; Montini E.; Aiuti A.; Mortellaro A. |
| Contributors: | Jofra Hernandez, R; Calabria, A; Sanvito, F; De Mattia, F; Farinelli, G; Scala, S; Visigalli, I; Carriglio, N; De Simone, M; Vezzoli, M; Cecere, F; Migliavacca, M; Basso-Ricci, L; Omrani, M; Benedicenti, F; Norata, R; Rancoita, P; Di Serio, C; Albertini, P; Cristofori, P; Naldini, L; Gentner, B; Montini, E; Aiuti, A; Mortellaro, A |
| Publisher Information: | Cell Press; US |
| Publication Year: | 2021 |
| Collection: | Università degli Studi di Milano-Bicocca: BOA (Bicocca Open Archive) |
| Subject Terms: | gene therapy; GLP; Good Laboratory Practice; inflammation; lentiviral vector; mouse model; myelodysplastic syndrome; X-linked chronic granulomatosis disease |
| Description: | 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. |
| Document Type: | article in journal/newspaper |
| File Description: | STAMPA |
| Language: | English |
| Relation: | info:eu-repo/semantics/altIdentifier/pmid/33010230; info:eu-repo/semantics/altIdentifier/wos/WOS:000607316600012; volume:29; issue:1; firstpage:86; lastpage:102; numberofpages:17; journal:MOLECULAR THERAPY; https://hdl.handle.net/10281/530490 |
| DOI: | 10.1016/j.ymthe.2020.09.030 |
| Availability: | https://hdl.handle.net/10281/530490; https://doi.org/10.1016/j.ymthe.2020.09.030 |
| Accession Number: | edsbas.897C6A09 |
| Database: | BASE |