The Optimized γ-Globin Lentiviral Vector GGHI-mB-3D Leads to Nearly Therapeutic HbF Levels In Vitro...
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
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Switzerland: MDPI AG
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English
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Switzerland: MDPI AG
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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 the disease phenotype in vitro. In the present st...
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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
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TN_cdi_doaj_primary_oai_doaj_org_article_c5506557ad3a4eb2ad4f6cda59415445
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_c5506557ad3a4eb2ad4f6cda59415445
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ISSN
1999-4915
E-ISSN
1999-4915
DOI
10.3390/v14122716
