Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to Treat β-thalassemia

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 ass...

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Bibliografski detalji
Glavni autori: Ronen, Keshet, Negre, Olivier, Roth, Shannah, Colomb, Charlotte, Malani, Nirav, Denaro, Maria, Brady, Troy, Fusil, Floriane, Gillet-Legrand, Beatrix, Hehir, Kathleen, Beuzard, Yves, Leboulch, Philippe, Down, Julian D, Payen, Emmanuel, Bushman, Frederic D
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2011
Teme:
Original Article
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3129560/
https://ncbi.nlm.nih.gov/pubmed/21386821
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.20
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