In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques

In utero gene therapy (IUGT) to the fetal hematopoietic compartment could be used to treat congenital blood disorders such as β-thalassemia. A humanised mouse model of β-thalassemia was used, in which heterozygous animals are anaemic with splenomegaly and extramedullary hematopoiesis. Intrahepatic i...

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Veröffentlicht in:Sci Rep
Hauptverfasser: Shangaris, Panicos, Loukogeorgakis, Stavros P., Subramaniam, Sindhu, Flouri, Christina, Jackson, Laurence H., Wang, Wei, Blundell, Michael P., Liu, Shanrun, Eaton, Simon, Bakhamis, Nahla, Ramachandra, Durrgah Latchumi, Maghsoudlou, Panayiotis, Urbani, Luca, Waddington, Simon N., Eddaoudi, Ayad, Archer, Joy, Antoniou, Michael N., Stuckey, Daniel J., Schmidt, Manfred, Thrasher, Adrian J., Ryan, Thomas M., De Coppi, Paolo, David, Anna L.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group UK 2019
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6690943/
https://ncbi.nlm.nih.gov/pubmed/31406195
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-019-48078-4
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