Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to T...
Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to Treat [beta]-thalassemia
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Milwaukee: Elsevier Limited
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Language
English
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Milwaukee: Elsevier Limited
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A lentiviral vector encoding β-globin flanked by insulator elements has been used to treat β-thalassemia (β-Thal) successfully in one human subject. However, a clonal expansion was observed after integration in the HMGA2 locus, raising the question of how commonly lentiviral integration would be associated with possible insertional activation. Here...
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Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to Treat [beta]-thalassemia
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TN_cdi_proquest_journals_1791990483
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_1791990483
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ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1038/mt.2011.20