Delivery on demand -- a new era of gene therapy?

Article Abstract:

An animal study published in 2002 showed that gene therapy may only work if the gene being introduced into the body is attached to a regulatory gene that controls the expression of the gene. In this case, it was the gene for erythropoietin, which stimulates the production of red blood cells. It was attached to a gene that responds to low levels of oxygen. When this happens, then and only then does the gene begin producing erythropoietin.

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Anemia and gene therapy-A matter of control

Article Abstract:

A means of stimulating the production of erythropoietin and its secretion into the bloodstream forms the basis of a treatment for anemia. A strategy that achieves this goal in a mouse model, is discussed where they used a genetically modified, antigen-specific B cell to supply erythropoietin to a mouse deficient in erythropoietin.

United Kingdom, Risk factors

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Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency

Article Abstract:

Gene therapy corrects inherited diseases, especially the deficiencies of hematopoietic cells. The genetic defect in X-linked severe combined immunodeficiency is corrected by the activation of the T-cell oncogen LMO2 using gene therapy.

United States, Dosage and administration, Hematopoietic stem cells, Oncogenes

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