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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Bibliografiset tiedot
Julkaisussa:Sci Rep
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: Nature Publishing Group UK 2019
Aiheet:
Article
Linkit: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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