K562 cells: a source for embryonic globin chains

Bhaumik, Kabita

doi:10.1016/0378-4347(91)80432-c

Cited by 2 publications

(2 citation statements)

References 22 publications

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“…The established K562 cell line derives from chronic myeloid leukemia cells. It is mostly triploid or tetraploid, typically including a triplicate of chromosome 11 [ 33 ], and cells produce γ-globin instead of β-globin protein even though the cell line is established from an adult patient. Therefore, they are appropriate cells for studying the expression of the transferred β-globin gene.…”

Section: Resultsmentioning

confidence: 99%

“…It was shown that the level of γ-globin mRNA far exceeded that of α-globin mRNA—fraction HBG/HBA—in non-transfected and transfected K562 cells, even at the 14th week of the long-term culture. This can be attributed to the nature of K562 cells, which are trisomic for chromosome 11 [ 33 ]. HBB mRNA, deriving mostly from the vector transgene, is a 0.13 fraction of the endogenous HBG mRNA at week 8, while the fraction HBB/HBG decreases from week 8 to week 14, as expected from Figure 4 Aa.…”

Section: Resultsmentioning

confidence: 99%

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Non-Viral Episomal Vector Mediates Efficient Gene Transfer of the β-Globin Gene into K562 and Human Haematopoietic Progenitor Cells

Lazaris,

Simantirakis,

Stavrou

et al. 2023

Genes

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β-Thalassemia is a subgroup of inherited blood disorders associated with mild to severe anemia with few and limited conventional therapy options. Lately, lentiviral vector-based gene therapy has been successfully applied for disease treatment. However, the current development of non-viral episomal vectors (EV), non-integrating and non-coding for viral proteins, may be helpful in generating valid alternatives to viral vectors. We constructed a non-viral, episomal vector pEPβ-globin for the physiological β-globin gene based on two human chromosomal elements: the scaffold or matrix attachment region (S/MAR), allowing for long nuclear retention and non-integration and the β-globin replication initiation region (IR), allowing for enhancement of replication and establishment. After nucleofections into K562 cells with a transfection efficiency of 24.62 ± 7.7%, the vector induces stable transfection and is detected in long-term cultures as a non-integrating, circular episome expressing the β-globin gene efficiently. Transfections into CD34+ cells demonstrate an average efficiency of 15.57 ± 11.64%. In the colony-forming cell assay, fluorescent colonies are 92.21%, which is comparable to those transfected with vector pEP-IR at 92.68%. Additionally, fluorescent colonies produce β-globin mRNA at a physiologically 3-fold higher level than the corresponding non-transfected cells. Vector pEPβ-globin provides the basis for the development of therapeutic EV for gene therapy of β-thalassemias.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%