| Gene therapy can be defined as the modification of the genome of cells to treat or prevent diseases. It is the most direct and effective method for the treatment of hereditary disorders. Hematopoietic stem cells (HSCs) are regarded as the most ideal target cells in gene therapy because of their capacity of self-renewing (being able to give rise to literally billions of progeny cells for essentially a lifetime) and pluripotent differentiation (being able to give rise to cells of all hematopoietic and lymphoid lineages). It is an attractive treatment strategy for many genetic and hematological diseases to transfer therapeutic genes into HSCs based on integrating vectors.β-thalassemia is a kind of common hereditary disease in hematopoietic system.. Till now, no satisfactory strategies can be performed effectively in the treatment of β-thalassemia. Human globin gene family resides in different loci, α-globin gene cluster and β-globin gene cluster. The function genes in both clusters can be expressed with the characteristics of erythroid-specificity, developmental stage-specificity and final product-balance. Once the balance between α -and β -globin final products was destroyed, thalassemia is coming. β-thalassemia results from deletions or mutations in regulatory regions or coding regions of β-globin gene cluster, which leads to the descendance or cessation of the synthesis of β-globin polypeptide, and thus the aggregation of redundant α -globin peptide.Viral vectors-mediated gene transfer to HSC are mostly used in the gene therapy of β-thalassemia by now. Viral vectors are efficient but currently appear to be too dangerous for routine clinical use, because their instability might cause unstable integration and expression of target gene. What's more, viral vectors may cause integration mutation during the procedure of gene transfer, and recombinant wild-type virus may be produced. So Gene delivery vehicles ("vectors") are the main...
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