The Optimized γ-Globin Lentiviral Vector GGHI-mB-3D Leads to Nearly Therapeutic HbF Levels In Vitro in CD34(+) Cells from Sickle Cell Disease Patients

We have previously demonstrated that both the original γ-globin lentiviral vector (LV) GGHI and the optimized GGHI-mB-3D LV, carrying the novel regulatory elements of the 3D HPFH-1 enhancer and the 3’ β-globin UTR, can significantly increase HbF production in thalassemic CD34(+) cells and ameliorate...

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Detalles Bibliográficos
Autores principales: Drakopoulou, Ekati, Georgomanoli, Maria, Lederer, Carsten W., Panetsos, Fottes, Kleanthous, Marina, Voskaridou, Ersi, Valakos, Dimitrios, Papanikolaou, Eleni, Anagnou, Nicholas P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9783242/
https://www.ncbi.nlm.nih.gov/pubmed/36560719
http://dx.doi.org/10.3390/v14122716

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9783242/
https://www.ncbi.nlm.nih.gov/pubmed/36560719
http://dx.doi.org/10.3390/v14122716

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