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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6690943/
https://www.ncbi.nlm.nih.gov/pubmed/31406195
http://dx.doi.org/10.1038/s41598-019-48078-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6690943/
https://www.ncbi.nlm.nih.gov/pubmed/31406195
http://dx.doi.org/10.1038/s41598-019-48078-4

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