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β-Globin Lentiviral Vectors Have Reduced Titers Due to Incomplete Vector RNA Genomes and Lowered Virion Production

ABSTRACT: β-Globin Lentiviral Vectors Have Reduced Titers Due to Incomplete Vector RNA Genomes and Lowered Virion Production

PROVIDER: PRJNA664591 | ENA |

REPOSITORIES: ENA

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β-Globin Lentiviral Vectors Have Reduced Titers Due to Incomplete Vector RNA Genomes and Lowered Virion Production

Project description:Lentiviral vectors (LV) commonly used for the treatment of hemoglobinopathies often have low titers and sub-optimal gene transfer efficiency for human hematopoietic stem and progenitor cells (HSPC), hindering clinical translation and commercialization for ex vivo gene therapy. We observed that a high percentage of β-globin LV viral genomic RNAs were incomplete towards the 3’ end in packaging cells and in released vector particles. The incomplete vector genomes impeded reverse transcription in target cells, limiting stable gene transfer to HSPC. By combining three modifications to vector design and production (shortening the vector length to 5.3-kb; expressing TAT protein during packaging; and packaging in PKR-/- cells) there was a 30-fold increase in vector titer and a 3-fold increase in vector infectivity in HSPC. These approaches may improve the manufacturing of β-globin and other complex LVs for enhanced gene delivery and may facilitate clinical applications.

2020-09-21 | GSE158252 | GEO

?-Globin Lentiviral Vectors Have Reduced Titers due to Incomplete Vector RNA Genomes and Lowered Virion Production.

Project description: Not available

| S-EPMC7897704 | biostudies-literature

Small RNA sequencing of human K562 cells transduced with lentiviral vectors overexpressing RUNX1

Project description:miRNA-Sequencing of RUNX1 overexpressing K562 cells were compared to K562 cells transduced with an empty vector control to analyse the differential expression of miRNAs in hematopoiesis. K562 cells were transduced either with an empty lentiviral vector as control or a lentiviral vector overexpressing human RUNX1. The samples were biological triplicates and the results were validated by qRT-PCR using SYBR green.

2016-06-01 | E-GEOD-70942 | biostudies-arrayexpress

Zebrafish Globin Locus

Project description:This SuperSeries is composed of the following subset Series: GSE35874: Zebrafish Globin Locus (ChIP-seq) GSE35875: Zebrafish Globin Locus (DNAse) Refer to individual Series

2012-02-17 | E-GEOD-35895 | biostudies-arrayexpress

Proteasome degradation of synthetic ubiquitinated globin

Project description:Analysis of the peptides resulting from proteasome-mediated degradation of K48-linked tetra ubiquitin conjugated to alpha globin

2021-08-24 | PXD004456 | Pride

Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small ?-Globin Locus Control Region Elements.

Project description: Not available

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Creating New ?-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences.

Project description: Not available

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Zebrafish Globin Locus

Project description:Zebrafish Globin Locus

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The pivotal and universal role of BGLT3 lncRNA in fetal globin gene expression

Project description:A highly selective and non-genotoxic G9a inhibitor, RK-701 was discovered, which upregulated the mRNA level of γ-globin but not β-globin both in human erythroid cells and in mice. Using RK-701, we examined the induction of the fetal (γ-) globin protein in human erythroid cells; HUDEP-2 and human CD34+ bone marrow and peripheral blood cells.

2023-03-11 | PXD033536 | Pride

SOX6 interacting proteins' identification in K562 cells for the regulation of γ-globin gene expression research

Project description:Human fetal γ-globin gene is developmentally silenced around the birth, and reactivation of γ-globin gene in adulthood sheds new light on ameliorating symptoms of hemoglobin disorders, such as sickle cell disease (SCD) and β-thalassemias. However, the precise regulation process of γ-globin remains incompletely understood. Here, we found that a new protein directly interacted with SOX6 and exerted a significant repression effect on the expression of γ-globin gene in erythroid cells. Further studies have demonstrated that it bound directly to γ-globin gene promoter via octamer binding motif, which in turn suppressed the transcriptional activity of γ-globin gene promoter. Thus, these data indicate that this new protein acts as a novel transcriptional repressor in the regulation of γ-globin gene expression through direct promoter binding, implying a potential alternative therapeutic target for the treatment of SCD and β-thalassemias.

2022-02-16 | PXD024259 | Pride

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